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These Bacteria Digest Food Waste Into Biodegradable Plastic

Factory Farm

Plastics have revolutionized manufacturing, but they’re still terrible for the environment.

Manufacturing plastics is an energy-intensive slog that ends in mountains of toxic industrial waste and greenhouse gas emissions. And then the plastic itself that we use ends up sitting in a garbage heap for thousands of years before it biodegrades.

Scientists have spent years investigating ways to manufacture plastics without ruining the planet, and a Toronto biotech startup called Genecis says it’s found a good answer: factories where vats of bacteria digest food waste and use it to form biodegradable plastic in their tiny microbial guts.

One-Two Punch

The plastic-pooping bacteria stand to clean up several kinds of pollution while churning out usable materials, according to Genecis.

That’s because the microbes feed on waste food or other organic materials — waste that CBC reported gives off 20 percent of Canada’s methane emissions as it sits in landfills.

Then What?

The plastic that the little buggers produce isn’t anything new. It’s called PHA and it’s used in anything that needs to biodegrade quickly, like those self-dissolving stitches. What’s new here is that food waste is much cheaper than the raw materials that usually go into plastics, leading Genecis to suspect it can make the same plastics for 40 percent less cost.

There are a lot of buzzworthy new alternative materials out there, but with a clear environmental and financial benefit, it’s possible these little bacteria factories might be here to stay.

READ MORE: Greener coffee pods? Bacteria help turn food waste into compostable plastic [CBC]

More on cleaning up plastics: The EU Just Voted to Completely ban Single-Use Plastics

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These Bacteria Digest Food Waste Into Biodegradable Plastic

You Can Now Preorder a $150,000 Hoverbike

Please, Santa?

It’s never too early to start writing your Christmas wish list, right? Because we know what’s now at the top of ours: a hoverbike.

We’ve had our eyes on Hoversurf’s Scorpion-3 since early last year — but now, the Russian drone start-up is accepting preorders on an updated version of the vehicle.

Flying Bike

The S3 2019 is part motorcycle and part quadcopter. According to the Hoversurf website, the battery-powered vehicle weighs 253 pounds and has a flight time of 10 to 25 minutes depending on operator weight. Its maximum legal speed is 60 mph — though as for how fast the craft can actually move, that’s unknown. Hoversurf also notes that the vehicle’s “safe flight altitude” is 16 feet, but again, we aren’t sure how high it can actually soar.

What we do know: The four blades that provide S3 with its lift spin at shin level, and while this certainly looks like it would be a safety hazard, the U.S. Department of Transportation’s Federal Aviation Administration approved the craft for legal use as an ultralight vehicle in September.

That means you can only operate an S3 for recreational or sports purposes — but you can’t cruise to work on your morning commute.

Plummeting Bank Account

You don’t need a pilot’s license to operate an S3, but you will need a decent amount of disposable income — the Star Wars-esque craft will set you back $150,000.

If that number doesn’t cause your eyes to cross, go ahead and slap down the $10,000 deposit needed to claim a spot in the reservation queue. You’ll then receive an email when it’s time to to place your order. You can expect to receive your S3 2019 two to six months after that, according to the company website.

That means there’s a pretty good chance you won’t be able to hover around your front yard this Christmas morning, but a 2019 jaunt is a genuine possibility.

READ MORE: For $150,000 You Can Now Order Your Own Hoverbike [New Atlas]

More on Hoversurf: Watch the World’s First Rideable Hoverbike in Flight

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You Can Now Preorder a $150,000 Hoverbike

FBI’s Tesla Criminal Probe Reportedly Centers on Model 3 Production

Ups and Downs

Can we please get off Mr. Musk’s Wild Ride now? We don’t know how much more of this Tesla rollercoaster we can take.

In 2018 alone, Elon Musk’s clean energy company has endured a faulty flufferbot, furious investors, and an SEC probe and settlement. But there was good news, too. Model 3 deliveries reportedly increased, and just this week, we found out that Tesla had a historic financial quarter, generating $312 million in profit.

And now we’re plummeting again.

Closing In

On Friday, The Wall Street Journal reported that the Federal Bureau of Investigation (FBI) is deepening a criminal probe into whether Tesla “misstated information about production of its Model 3 sedans and misled investors about the company’s business going back to early 2017.”

We’ve known about the FBI’s Tesla criminal probe since September 18, but this is the first report confirming that Model 3 production is at the center of the investigation.

According to the WSJ’s sources, FBI agents have been reaching out to former Tesla employees in recent weeks to ask if they’d be willing to testify in the criminal case, though no word yet on whether any have agreed.

Casual CEO

We might be having trouble keeping up with these twists and turns, but Musk seems to be taking the FBI’s Tesla criminal probe all in stride — he spent much of Friday afternoon joking around with his Twitter followers about dank memes.

Clearly he has the stomach for this, but it’d be hard to blame any Tesla investors for deciding they’d had enough.

READ MORE: Tesla Faces Deepening Criminal Probe Over Whether It Misstated Production Figures [The Wall Street Journal]

More on Tesla: Elon Musk Says Your Tesla Will Earn You Money While You Sleep

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FBI’s Tesla Criminal Probe Reportedly Centers on Model 3 Production

Zero Gravity Causes Worrisome Changes In Astronauts’ Brains

Danger, Will Robinson

As famous Canadian astronaut Chris Hadfield demonstrated with his extraterrestrial sob session, fluids behave strangely in space.

And while microgravity makes for a great viral video, it also has terrifying medical implications that we absolutely need to sort out before we send people into space for the months or years necessary for deep space exploration.

Specifically, research published Thursday In the New England Journal of Medicine demonstrated that our brains undergo lasting changes after we spend enough time in space. According to the study, cerebrospinal fluid — which normally cushions our brain and spinal cord — behaves differently in zero gravity, causing it to pool around and squish our brains.

Mysterious Symptoms

The brains of the Russian cosmonauts who were studied in the experiment mostly bounced back upon returning to Earth.

But even seven months later, some abnormalities remained. According to National Geographic, the researchers suspect that high pressure  inside the cosmonauts’ skulls may have squeezed extra water into brain cells which later drained out en masse.

Now What?

So far, scientists don’t know whether or not this brain shrinkage is related to any sort of cognitive or other neurological symptoms — it might just be a weird quirk of microgravity.

But along with other space hazards like deadly radiation and squished eyeballs, it’s clear that we have a plethora of medical questions to answer before we set out to explore the stars.

READ MORE: Cosmonaut brains show space travel causes lasting changes [National Geographic]

More on space medicine: Traveling to Mars Will Blast Astronauts With Deadly Cosmic Radiation, new Data Shows

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Zero Gravity Causes Worrisome Changes In Astronauts’ Brains

We Aren’t Growing Enough Healthy Foods to Feed Everyone on Earth

Check Yourself

The agriculture industry needs to get its priorities straight.

According to a newly published study, the world food system is producing too many unhealthy foods and not enough healthy ones.

“We simply can’t all adopt a healthy diet under the current global agriculture system,” said study co-author Evan Fraser in a press release. “Results show that the global system currently overproduces grains, fats, and sugars, while production of fruits and vegetables and, to a smaller degree, protein is not sufficient to meet the nutritional needs of the current population.”

Serving Downsized

For their study, published Tuesday in the journal PLOS ONE, researchers from the University of Guelph compared global agricultural production with consumption recommendations from Harvard University’s Healthy Eating Plate guide. Their findings were stark: The agriculture industry’s overall output of healthy foods does not match humanity’s needs.

Instead of the recommended eight servings of grains per person, it produces 12. And while nutritionists recommend we each consume 15 servings of fruits and vegetables daily, the industry produces just five. The mismatch continues for oils and fats (three servings instead of one), protein (three servings instead of five), and sugar (four servings when we don’t need any).

Overly Full Plate

The researchers don’t just point out the problem, though — they also calculated what it would take to address the lack of healthy foods while also helping the environment.

“For a growing population, our calculations suggest that the only way to eat a nutritionally balanced diet, save land, and reduce greenhouse gas emission is to consume and produce more fruits and vegetables as well as transition to diets higher in plant-based protein,” said Fraser.

A number of companies dedicated to making plant-based proteins mainstream are already gaining traction. But unfortunately, it’s unlikely that the agriculture industry will decide to prioritize growing fruits and veggies over less healthy options as long as people prefer having the latter on their plates.

READ MORE: Not Enough Fruits, Vegetables Grown to Feed the Planet, U of G Study Reveals [University of Guelph]

More on food scarcity: To Feed a Hungry Planet, We’re All Going to Need to Eat Less Meat

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We Aren’t Growing Enough Healthy Foods to Feed Everyone on Earth

Report Identifies China as the Source of Ozone-Destroying Emissions

Emissions Enigma

For years, a mystery puzzled environmental scientists. The world had banned the use of many ozone-depleting compounds in 2010. So why were global emission levels still so high?

The picture started to clear up in June. That’s when The New York Times published an investigation into the issue. China, the paper claimed, was to blame for these mystery emissions. Now it turns out the paper was probably right to point a finger.

Accident or Incident

In a paper published recently in the journal Geophysical Research Letters, an international team of researchers confirms that eastern China is the source of at least half of the 40,000 tonnes of carbon tetrachloride emissions currently entering the atmosphere each year.

They figured this out using a combination of ground-based and airborne atmospheric concentration data from near the Korean peninsula. They also relied on two models that simulated how the gases would move through the atmosphere.

Though they were able to narrow down the source to China, the researchers weren’t able to say exactly who’s breaking the ban and whether they even know about the damage they’re doing.

Pinpoint

“Our work shows the location of carbon tetrachloride emissions,” said co-author Matt Rigby in a press release. “However, we don’t yet know the processes or industries that are responsible. This is important because we don’t know if it is being produced intentionally or inadvertently.”

If we can pinpoint the source of these emissions, we can start working on stopping them and healing our ozone. And given that we’ve gone nearly a decade with minimal progress on that front, there’s really no time to waste.

READ MORE: Location of Large ‘Mystery’ Source of Banned Ozone Depleting Substance Uncovered [University of Bristol]

More on carbon emissions: China Has (Probably) Been Pumping a Banned Gas Into the Atmosphere

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Report Identifies China as the Source of Ozone-Destroying Emissions

There’s No Way China’s Artificial Moon Will Work, Says Expert

Good Luck

On October 10, a Chinese organization called the Tian Fu New Area Science Society revealed plans to replace the streetlights in the city of Chengdu with a satellite designed to reflect sunlight toward the Earth’s surface at night.

But in a new interview with Astronomy, an associate professor of aerospace engineering at the University of Texas at Austin named Ryan Russel argued that based on what he’s read, the artificial moon plan would be impossible to implement.

Promised the Moon

Wu Chunfeng, the head of the Tian Fu New Area Science Society, told China Daily the artificial moon would orbit about 310 miles above Earth, delivering an expected brightness humans would perceive to be about one-fifth that of a typical streetlight.

The plan is to launch one artificial moon in 2020 and then three more in 2022 if the first works as hoped. Together, these satellites could illuminate an area of up to 4,000 square miles, Chunfeng claims.

But Russell is far from convinced.

“Their claim for 1 [low-earth orbit satellite] at [300 miles] must be a typo or misinformed spokesperson,” he told Astronomy. “The article I read implied you could hover a satellite over a particular city, which of course is not possible.”

Overkill Overhead

To keep the satellite in place over Chengdu, it would need to be about 22,000 miles above the Earth’s surface, said Russel, and its reflective surface would need to be massive to reflect sunlight from that distance. At an altitude of just 300 miles, the satellite would quickly zip around the Earth, constantly illuminating new locations.

Even if the city could put the artificial moon plan into action, though, Russell isn’t convinced it should.

“It’s a very complicated solution that affects everyone to a simple problem that affects a few,” he told Astronomy. “It’s light pollution on steroids.”

Maybe Chengdu shouldn’t give up on its streetlights just yet.

READ MORE: Why China’s Artificial Moon Probably Won’t Work [Astronomy]

More on the artificial moon: A Chinese City Plans to Replace Its Streetlights With an Artificial Moon

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There’s No Way China’s Artificial Moon Will Work, Says Expert

Clean Coal Startup Turns Human Waste Into Earth-Friendly Fuel

Gold Nuggets

A company called Ingelia says it’s figured out a way to turn human waste — the solid kind — into a combustible material it’s calling biochar. And if Ingelia’s claims are accurate, biochar can be burned for fuel just like coalexcept with nearzero greenhouse gas emissions, according to Business Insider.

That’s because almost all of the pollutants and more harmful chemicals that would normally be given off while burning solid fuels is siphoned away into treatable liquid waste, leaving a dry, combustible rod of poop fuel.

“Clean Coal

Ingelia, which is currently working to strike a deal with Spanish waste management facilities, hopes to make enough biochar to replace 220 thousand tons of coal per year, corresponding to 500 thousand tons of carbon dioxide emissions.

But that’s by 2022, at which point we’ll have even less time to reach the urgent clean energy goals of that doomsday United Nations report. In an ideal world, we would have moved away from coal years ago. At least this gives us a viable alternative as we transition to other, renewable forms of electricity.

So while we can, in part, poop our way to a better world, biochar — and other new sewage-based energy sources — will only be one of many new world-saving sources of clean energy.

READ MORE: This Spanish company found a way to produce a fuel that emits no CO2 — and it’s made of sewage [Business Insider]

More on poop: Edible Tech is Finally Useful, is Here to Help you Poop

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Clean Coal Startup Turns Human Waste Into Earth-Friendly Fuel

Ford’s Self-Driving Cars Are About to Chauffeur Your Senator

Green-Light District

It doesn’t matter how advanced our self-driving cars get — if they aren’t allowed on roads, they aren’t going to save any lives.

The future of autonomous vehicles (AVs) in the U.S. depends on how lawmakers in Washington D.C. choose to regulate the vehicles. But until now, AV testing has largely taken place far from the nation’s capital, mostly in California and Arizona.

Ford is about to change that. The company just announced plans to be the first automaker to test its self-driving cars in the Distinct of Columbia — and how lawmakers feel about those vehicles could influence future AV legislation.

Career Day

Sherif Marakby, CEO of Ford Autonomous Vehicles, announced the decision to begin testing in D.C. via a blog post last week. According to Marakby, Ford’s politician-friendly focus will be on figuring out how its AVs could promote job creation in the District.

To that end, Ford plans to assess how AVs could increase mobility in D.C., thereby helping residents get to jobs that might otherwise be outside their reach, as well as train residents for future positions as AV technicians or operators.

Up Close and Personal

Marakby notes that D.C. is a particularly suitable location for this testing because the District is usually bustling with activity. The population increases significantly during the day as commuters arrive from the suburbs for work, while millions of people flock to D.C. each year for conferences or tourism.

D.C. is also home to the people responsible for crafting and passing AV legislation. “[I]t’s important that lawmakers see self-driving vehicles with their own eyes as we keep pushing for legislation that governs their safe use across the country,” Marakby wrote.

Ford’s ultimate goal is to launch a commercial AV service in D.C. in 2021. With this testing, the company has the opportunity to directly influence the people who could help it reach that goal — or oppose it.

READ MORE: A Monumental Moment: Our Self-Driving Business Development Expands to Washington, D.C. [Medium]

More on AV legislation: U.S. Senators Reveal the Six Principles They’ll Use to Regulate Self-Driving Vehicles

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Ford’s Self-Driving Cars Are About to Chauffeur Your Senator

WHO Director: Air Pollution Is the “New Tobacco”

Wrong Direction

Breathing polluted air is as likely to kill you as tobacco use — worldwide, each kills about 7 million people annually. But while the world is making progress in the war against tobacco, air pollution is getting worse.

The Director General of the World Health Organization (WHO) hopes to change that.

“The world has turned the corner on tobacco,” wrote Tedros Adhanom Ghebreyesus in an opinion piece published by The Guardian on Saturday. “Now it must do the same for the ‘new tobacco’ — the toxic air that billions breathe every day.”

Taking Action

According to the WHO, nine out of 10 people in the world breathe polluted air.

This week, the organization is hosting the first Global Conference on Air Pollution and Health, and Ghebreyesus is hopeful world leaders will use the conference as the opportunity to commit to cutting air pollution in their nations.

“Despite the overwhelming evidence, political action is still urgently needed to boost investments and speed up action to reduce air pollution,” he wrote, noting that this action could take the form of more stringent air quality standards, improved access to clean energy, or increased investment in green technologies.

Reduced Risk

The impact sustained action against air pollution could have on public health is hard to overstate.

“No one, rich or poor, can escape air pollution. A clean and healthy environment is the single most important precondition for ensuring good health,” wrote Ghebreyesus in his Guardian piece. “By cleaning up the air we breathe, we can prevent or at least reduce some of the greatest health risks.”

The conference ends on Thursday, so we won’t have to wait long to see which nations do — or don’t — heed the WHO’s call to action.

READ MORE: Air Pollution Is the New Tobacco. Time to Tackle This Epidemic [The Guardian]

More on air pollution: Dumber Humans — That’s Just One Effect of a More Polluted Future

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WHO Director: Air Pollution Is the “New Tobacco”

Scientists May Have Put Microbes in a State of Quantum Entanglement

Hall of Mirrors

A few years ago, the journal Small published a study showing how photosynthetic bacteria could absorb and release photons as the light bounced across a minuscule gap between two mirrors.

Now, a retroactive look at the study’s data published in The Journal of Physics Communications suggests something more may have been going on. The bacteria may have been the first living organisms to operate in the realm of quantum physics, becoming entangled with the bouncing light at the quantum scale.

Cat’s Cradle

The experiment in question, as described by Scientific American, involved individual photons — the smallest quantifiable unit of light that can behave like a tiny particle but also a wave of energy within quantum physics — bouncing between two mirrors separated by a microscopic distance.

But a look at the energy levels in the experimental setup suggests that the bacteria may have become entangled, as some individual photons seem to have simultaneously interacted with and missed the bacterium at the same time.

Super Position

There’s reason to be skeptical of these results until someone actually recreates the experiment while looking for signs of quantum interactions. As with any look back at an existing study, scientists are restricted to the amount and quality of data that was already published. And, as Scientific American noted, the energy levels of the bacteria and the mirror setup should have been recorded individually — which they were not — in order to verify quantum entanglement.

But if this research holds up, it would be the first time a life form operated on the realm of quantum physics, something usually limited to subatomic particles. And even though the microbes are small, that’s a big deal.

READ MORE“Schrödinger’s Bacterium” Could Be a Quantum Biology Milestone [Scientific American]

More on quantum physics: The World’s First Practical Quantum Computer May Be Just Five Years Away

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Scientists May Have Put Microbes in a State of Quantum Entanglement

Gene Therapy | Pfizer: One of the world’s premier …

Gene therapy is a technology aimed at correcting or fixing a gene that may be defective. This exciting and potentially transformative area of research is focused on the development of potential treatments for monogenic diseases, or diseases that are caused by a defect in one gene.

The technology involves the introduction of genetic material (DNA or RNA) into the body, often through delivering a corrected copy of a gene to a patients cells to compensate for a defective one, using a viral vector.

The technology involves the introduction of genetic material (DNA or RNA) into the body, often through delivering a corrected copy of a gene to a patients cells to compensate for a defective one, using a viral vector.

Viral vectors can be developed using adeno-associated virus (AAV), a naturally occurring virus which has been adapted for gene therapy use. Its ability to deliver genetic material to a wide range of tissues makes AAV vectors useful for transferring therapeutic genes into target cells. Gene therapy research holds tremendous promise in leading to the possible development of highly-specialized, potentially one-time delivery treatments for patients suffering from rare, monogenic diseases.

Pfizer aims to build an industry-leading gene therapy platform with a strategy focused on establishing a transformational portfolio through in-house capabilities, and enhancing those capabilities through strategic collaborations, as well as potential licensing and M&A activities.

We’re working to access the most effective vector designs available to build a robust clinical stage portfolio, and employing a scalable manufacturing approach, proprietary cell lines and sophisticated analytics to support clinical development.

In addition, we’re collaborating with some of the foremost experts in this field, through collaborations with Spark Therapeutics, Inc., on a potentially transformative gene therapy treatment for hemophilia B, which received Breakthrough Therapy designation from the US Food and Drug Administration, and 4D Molecular Therapeutics to discover and develop targeted next-generation AAV vectors for cardiac disease.

Gene therapy holds the promise of bringing true disease modification for patients suffering from devastating diseases, a promise were working to seeing become a reality in the years to come.

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Gene Therapy | Pfizer: One of the world’s premier …

Gene therapy | Cancer in general | Cancer Research UK

Gene therapy is a cancer treatment that is still in the early stages of research.

Genes are coded messages that tell cells how to make proteins. Proteins are the molecules that control the way cells behave. Our genes decide what we look like and how our body works.We have many thousands of separate genes.

Genes are made ofDNAand they are in the nucleus of the cell. The nucleus is the cell’s control centre.Genes are grouped together to make chromosomes. We inherit half our chromosomes from our mother and half from our father.

Cancer cells are different from normal cells. They have changes (called faults or mutations) in several of their genes which make them divide too often and form a tumour. The genes that are damaged mightbe:

Many gene changes thatmake a cell become cancerous are caused by environmental or lifestyle factors. A small numberof people haveinherited faulty genes that increase their risk of particular types of cancer.

Gene therapy is a type of treatment which uses genes to treat illnesses. Researchers have been developing differenttypes of gene therapyto treat cancer.

The ideas for these new treatments have come about because we are beginning to understand how cancer cells are different from normal cells. It is stillearly days for this type of treatment. Some of these treatments are being looked at in clinical trials. Otherscan now be used for some people with types of cancer such as melanoma skin cancer.

Getting genes into cancer cells is one of the most difficult aspects of gene therapy. Researchers are working on finding new and better ways of doing this. The gene is usually taken into the cancer cell by a carrier called a vector.

The most common types of carrier used in gene therapy are viruses because they can enter cells and deliver genetic material. The viruses have been changed so that they cannot cause serious disease but they may still cause mild, flu-like symptoms.

Some viruses have been changed in the laboratory so that they target cancer cells and not healthy cells. So they only carry the gene into cancer cells.

Researchers are testing other types of carrier such as inactivated bacteria.

Researchers are looking at different ways of using gene therapy:

Some types of gene therapy aim to boost the body’s natural ability to attack cancer cells. Ourimmune systemhas cells that recognise and kill harmful things that can cause disease, such as cancer cells.

There are many different types of immune cell. Some of them produce proteins that encourage other immune cells to destroy cancer cells. Some types of therapy add genes to a patient’s immune cells. Thismakes them better at finding or destroying particular types of cancer.

There are a few trials using this type of gene therapy in the UK.

Some gene therapies put genes into cancer cells to make the cells more sensitive to particular treatments. The aim is to make treatments,such as chemotherapy or radiotherapy, work better.

Some types of gene therapy deliver genes into the cancer cells that allow the cells to change drugs from an inactive form to an active form. The inactive form of the drug is called a pro drug.

First of all you have treatment with thecarrier containing the gene, then you havethe pro drug.The pro drug circulates in the body and doesn’t harm normal cells. But when it reaches the cancer cells, it is activated by the gene and the drug kills the cancer cells.

Some gene therapies block processes that cancer cells use to survive. For example, most cells in the body are programmed to die if their DNA is damaged beyond repair. This is called programmed cell death or apoptosis. Cancer cells block this process so they don’t die even when they are supposed to.

Some gene therapy strategies aim to reverse this blockage. Researchers are looking at whetherthese new types of treatment will make the cancer cells die.

Some viruses infect and kill cells. Researchers are working on ways to change these viruses so they only target and kill cancer cells, leaving healthy cells alone.

This sort of treatment uses the viruses to kill cancer cells directly rather than to deliver genes. So it is not cancer gene therapy in the true sense of the word. But doctors sometimes refer to it as gene therapy.

An example is a drug called T-VEC (talimogene laherparepvec), also known as Imlygic. It uses a strain of the cold sore virus (herpes simplex virus) that has been changed by altering the genes that tell the virus how to behave. It tells the virus to destroy the cancer cells and ignore the healthy cells.

T-VEC is now available as a treatment for melanoma skin cancer. It can be used to treat some people with melanomawhose cancer cannot be removed with surgery. It is also being looked at in trials for head and neck cancer. You have T-VEC as an injection directly into the melanoma or head and neck cancer.

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Gene therapy | Cancer in general | Cancer Research UK

Gene therapy – Mayo Clinic

Overview

Gene therapy involves altering the genes inside your body’s cells in an effort to treat or stop disease.

Genes contain your DNA the code that controls much of your body’s form and function, from making you grow taller to regulating your body systems. Genes that don’t work properly can cause disease.

Gene therapy replaces a faulty gene or adds a new gene in an attempt to cure disease or improve your body’s ability to fight disease. Gene therapy holds promise for treating a wide range of diseases, such as cancer, cystic fibrosis, heart disease, diabetes, hemophilia and AIDS.

Researchers are still studying how and when to use gene therapy. Currently, in the United States, gene therapy is available only as part of a clinical trial.

Gene therapy is used to correct defective genes in order to cure a disease or help your body better fight disease.

Researchers are investigating several ways to do this, including:

Gene therapy has some potential risks. A gene can’t easily be inserted directly into your cells. Rather, it usually has to be delivered using a carrier, called a vector.

The most common gene therapy vectors are viruses because they can recognize certain cells and carry genetic material into the cells’ genes. Researchers remove the original disease-causing genes from the viruses, replacing them with the genes needed to stop disease.

This technique presents the following risks:

The gene therapy clinical trials underway in the U.S. are closely monitored by the Food and Drug Administration and the National Institutes of Health to ensure that patient safety issues are a top priority during research.

Currently, the only way for you to receive gene therapy is to participate in a clinical trial. Clinical trials are research studies that help doctors determine whether a gene therapy approach is safe for people. They also help doctors understand the effects of gene therapy on the body.

Your specific procedure will depend on the disease you have and the type of gene therapy being used.

For example, in one type of gene therapy:

Viruses aren’t the only vectors that can be used to carry altered genes into your body’s cells. Other vectors being studied in clinical trials include:

The possibilities of gene therapy hold much promise. Clinical trials of gene therapy in people have shown some success in treating certain diseases, such as:

But several significant barriers stand in the way of gene therapy becoming a reliable form of treatment, including:

Gene therapy continues to be a very important and active area of research aimed at developing new, effective treatments for a variety of diseases.

Explore Mayo Clinic studies testing new treatments, interventions and tests as a means to prevent, detect, treat or manage this disease.

Dec. 29, 2017

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Gene therapy – Mayo Clinic

Gene therapy – Wikipedia

In the medicine field, gene therapy (also called human gene transfer) is the therapeutic delivery of nucleic acid into a patient’s cells as a drug to treat disease.[1][2] The first attempt at modifying human DNA was performed in 1980 by Martin Cline, but the first successful nuclear gene transfer in humans, approved by the National Institutes of Health, was performed in May 1989.[3] The first therapeutic use of gene transfer as well as the first direct insertion of human DNA into the nuclear genome was performed by French Anderson in a trial starting in September 1990.

Between 1989 and February 2016, over 2,300 clinical trials had been conducted, more than half of them in phase I.[4]

Not all medical procedures that introduce alterations to a patient’s genetic makeup can be considered gene therapy. Bone marrow transplantation and organ transplants in general have been found to introduce foreign DNA into patients.[5] Gene therapy is defined by the precision of the procedure and the intention of direct therapeutic effect.

Gene therapy was conceptualized in 1972, by authors who urged caution before commencing human gene therapy studies.

The first attempt, an unsuccessful one, at gene therapy (as well as the first case of medical transfer of foreign genes into humans not counting organ transplantation) was performed by Martin Cline on 10 July 1980.[6][7] Cline claimed that one of the genes in his patients was active six months later, though he never published this data or had it verified[8] and even if he is correct, it’s unlikely it produced any significant beneficial effects treating beta-thalassemia.

After extensive research on animals throughout the 1980s and a 1989 bacterial gene tagging trial on humans, the first gene therapy widely accepted as a success was demonstrated in a trial that started on 14 September 1990, when Ashi DeSilva was treated for ADA-SCID.[9]

The first somatic treatment that produced a permanent genetic change was performed in 1993.[citation needed]

Gene therapy is a way to fix a genetic problem at its source. The polymers are either translated into proteins, interfere with target gene expression, or possibly correct genetic mutations.

The most common form uses DNA that encodes a functional, therapeutic gene to replace a mutated gene. The polymer molecule is packaged within a “vector”, which carries the molecule inside cells.

Early clinical failures led to dismissals of gene therapy. Clinical successes since 2006 regained researchers’ attention, although as of 2014[update], it was still largely an experimental technique.[10] These include treatment of retinal diseases Leber’s congenital amaurosis[11][12][13][14] and choroideremia,[15] X-linked SCID,[16] ADA-SCID,[17][18] adrenoleukodystrophy,[19] chronic lymphocytic leukemia (CLL),[20] acute lymphocytic leukemia (ALL),[21] multiple myeloma,[22] haemophilia,[18] and Parkinson’s disease.[23] Between 2013 and April 2014, US companies invested over $600 million in the field.[24]

The first commercial gene therapy, Gendicine, was approved in China in 2003 for the treatment of certain cancers.[25] In 2011 Neovasculgen was registered in Russia as the first-in-class gene-therapy drug for treatment of peripheral artery disease, including critical limb ischemia.[26]In 2012 Glybera, a treatment for a rare inherited disorder, became the first treatment to be approved for clinical use in either Europe or the United States after its endorsement by the European Commission.[10][27]

Following early advances in genetic engineering of bacteria, cells, and small animals, scientists started considering how to apply it to medicine. Two main approaches were considered replacing or disrupting defective genes.[28] Scientists focused on diseases caused by single-gene defects, such as cystic fibrosis, haemophilia, muscular dystrophy, thalassemia, and sickle cell anemia. Glybera treats one such disease, caused by a defect in lipoprotein lipase.[27]

DNA must be administered, reach the damaged cells, enter the cell and either express or disrupt a protein.[29] Multiple delivery techniques have been explored. The initial approach incorporated DNA into an engineered virus to deliver the DNA into a chromosome.[30][31] Naked DNA approaches have also been explored, especially in the context of vaccine development.[32]

Generally, efforts focused on administering a gene that causes a needed protein to be expressed. More recently, increased understanding of nuclease function has led to more direct DNA editing, using techniques such as zinc finger nucleases and CRISPR. The vector incorporates genes into chromosomes. The expressed nucleases then knock out and replace genes in the chromosome. As of 2014[update] these approaches involve removing cells from patients, editing a chromosome and returning the transformed cells to patients.[33]

Gene editing is a potential approach to alter the human genome to treat genetic diseases,[34] viral diseases,[35] and cancer.[36] As of 2016[update] these approaches were still years from being medicine.[37][38]

Gene therapy may be classified into two types:

In somatic cell gene therapy (SCGT), the therapeutic genes are transferred into any cell other than a gamete, germ cell, gametocyte, or undifferentiated stem cell. Any such modifications affect the individual patient only, and are not inherited by offspring. Somatic gene therapy represents mainstream basic and clinical research, in which therapeutic DNA (either integrated in the genome or as an external episome or plasmid) is used to treat disease.

Over 600 clinical trials utilizing SCGT are underway[when?] in the US. Most focus on severe genetic disorders, including immunodeficiencies, haemophilia, thalassaemia, and cystic fibrosis. Such single gene disorders are good candidates for somatic cell therapy. The complete correction of a genetic disorder or the replacement of multiple genes is not yet possible. Only a few of the trials are in the advanced stages.[39]

In germline gene therapy (GGT), germ cells (sperm or egg cells) are modified by the introduction of functional genes into their genomes. Modifying a germ cell causes all the organism’s cells to contain the modified gene. The change is therefore heritable and passed on to later generations. Australia, Canada, Germany, Israel, Switzerland, and the Netherlands[40] prohibit GGT for application in human beings, for technical and ethical reasons, including insufficient knowledge about possible risks to future generations[40] and higher risks versus SCGT.[41] The US has no federal controls specifically addressing human genetic modification (beyond FDA regulations for therapies in general).[40][42][43][44]

The delivery of DNA into cells can be accomplished by multiple methods. The two major classes are recombinant viruses (sometimes called biological nanoparticles or viral vectors) and naked DNA or DNA complexes (non-viral methods).

In order to replicate, viruses introduce their genetic material into the host cell, tricking the host’s cellular machinery into using it as blueprints for viral proteins. Retroviruses go a stage further by having their genetic material copied into the genome of the host cell. Scientists exploit this by substituting a virus’s genetic material with therapeutic DNA. (The term ‘DNA’ may be an oversimplification, as some viruses contain RNA, and gene therapy could take this form as well.) A number of viruses have been used for human gene therapy, including retroviruses, adenoviruses, herpes simplex, vaccinia, and adeno-associated virus.[4] Like the genetic material (DNA or RNA) in viruses, therapeutic DNA can be designed to simply serve as a temporary blueprint that is degraded naturally or (at least theoretically) to enter the host’s genome, becoming a permanent part of the host’s DNA in infected cells.

Non-viral methods present certain advantages over viral methods, such as large scale production and low host immunogenicity. However, non-viral methods initially produced lower levels of transfection and gene expression, and thus lower therapeutic efficacy. Later technology remedied this deficiency.[citation needed]

Methods for non-viral gene therapy include the injection of naked DNA, electroporation, the gene gun, sonoporation, magnetofection, the use of oligonucleotides, lipoplexes, dendrimers, and inorganic nanoparticles.

Some of the unsolved problems include:

Three patients’ deaths have been reported in gene therapy trials, putting the field under close scrutiny. The first was that of Jesse Gelsinger, who died in 1999 because of immune rejection response.[51] One X-SCID patient died of leukemia in 2003.[9] In 2007, a rheumatoid arthritis patient died from an infection; the subsequent investigation concluded that the death was not related to gene therapy.[52]

In 1972 Friedmann and Roblin authored a paper in Science titled “Gene therapy for human genetic disease?”[53] Rogers (1970) was cited for proposing that exogenous good DNA be used to replace the defective DNA in those who suffer from genetic defects.[54]

In 1984 a retrovirus vector system was designed that could efficiently insert foreign genes into mammalian chromosomes.[55]

The first approved gene therapy clinical research in the US took place on 14 September 1990, at the National Institutes of Health (NIH), under the direction of William French Anderson.[56] Four-year-old Ashanti DeSilva received treatment for a genetic defect that left her with ADA-SCID, a severe immune system deficiency. The defective gene of the patient’s blood cells was replaced by the functional variant. Ashantis immune system was partially restored by the therapy. Production of the missing enzyme was temporarily stimulated, but the new cells with functional genes were not generated. She led a normal life only with the regular injections performed every two months. The effects were successful, but temporary.[57]

Cancer gene therapy was introduced in 1992/93 (Trojan et al. 1993).[58] The treatment of glioblastoma multiforme, the malignant brain tumor whose outcome is always fatal, was done using a vector expressing antisense IGF-I RNA (clinical trial approved by NIH protocolno.1602 November 24, 1993,[59] and by the FDA in 1994). This therapy also represents the beginning of cancer immunogene therapy, a treatment which proves to be effective due to the anti-tumor mechanism of IGF-I antisense, which is related to strong immune and apoptotic phenomena.

In 1992 Claudio Bordignon, working at the Vita-Salute San Raffaele University, performed the first gene therapy procedure using hematopoietic stem cells as vectors to deliver genes intended to correct hereditary diseases.[60] In 2002 this work led to the publication of the first successful gene therapy treatment for adenosine deaminase deficiency (ADA-SCID). The success of a multi-center trial for treating children with SCID (severe combined immune deficiency or “bubble boy” disease) from 2000 and 2002, was questioned when two of the ten children treated at the trial’s Paris center developed a leukemia-like condition. Clinical trials were halted temporarily in 2002, but resumed after regulatory review of the protocol in the US, the United Kingdom, France, Italy, and Germany.[61]

In 1993 Andrew Gobea was born with SCID following prenatal genetic screening. Blood was removed from his mother’s placenta and umbilical cord immediately after birth, to acquire stem cells. The allele that codes for adenosine deaminase (ADA) was obtained and inserted into a retrovirus. Retroviruses and stem cells were mixed, after which the viruses inserted the gene into the stem cell chromosomes. Stem cells containing the working ADA gene were injected into Andrew’s blood. Injections of the ADA enzyme were also given weekly. For four years T cells (white blood cells), produced by stem cells, made ADA enzymes using the ADA gene. After four years more treatment was needed.[62]

Jesse Gelsinger’s death in 1999 impeded gene therapy research in the US.[63][64] As a result, the FDA suspended several clinical trials pending the reevaluation of ethical and procedural practices.[65]

The modified cancer gene therapy strategy of antisense IGF-I RNA (NIH n 1602)[59] using antisense / triple helix anti-IGF-I approach was registered in 2002 by Wiley gene therapy clinical trial – n 635 and 636. The approach has shown promising results in the treatment of six different malignant tumors: glioblastoma, cancers of liver, colon, prostate, uterus, and ovary (Collaborative NATO Science Programme on Gene Therapy USA, France, Poland n LST 980517 conducted by J. Trojan) (Trojan et al., 2012). This anti-gene antisense/triple helix therapy has proven to be efficient, due to the mechanism stopping simultaneously IGF-I expression on translation and transcription levels, strengthening anti-tumor immune and apoptotic phenomena.

Sickle-cell disease can be treated in mice.[66] The mice which have essentially the same defect that causes human cases used a viral vector to induce production of fetal hemoglobin (HbF), which normally ceases to be produced shortly after birth. In humans, the use of hydroxyurea to stimulate the production of HbF temporarily alleviates sickle cell symptoms. The researchers demonstrated this treatment to be a more permanent means to increase therapeutic HbF production.[67]

A new gene therapy approach repaired errors in messenger RNA derived from defective genes. This technique has the potential to treat thalassaemia, cystic fibrosis and some cancers.[68]

Researchers created liposomes 25 nanometers across that can carry therapeutic DNA through pores in the nuclear membrane.[69]

In 2003 a research team inserted genes into the brain for the first time. They used liposomes coated in a polymer called polyethylene glycol, which unlike viral vectors, are small enough to cross the bloodbrain barrier.[70]

Short pieces of double-stranded RNA (short, interfering RNAs or siRNAs) are used by cells to degrade RNA of a particular sequence. If a siRNA is designed to match the RNA copied from a faulty gene, then the abnormal protein product of that gene will not be produced.[71]

Gendicine is a cancer gene therapy that delivers the tumor suppressor gene p53 using an engineered adenovirus. In 2003, it was approved in China for the treatment of head and neck squamous cell carcinoma.[25]

In March researchers announced the successful use of gene therapy to treat two adult patients for X-linked chronic granulomatous disease, a disease which affects myeloid cells and damages the immune system. The study is the first to show that gene therapy can treat the myeloid system.[72]

In May a team reported a way to prevent the immune system from rejecting a newly delivered gene.[73] Similar to organ transplantation, gene therapy has been plagued by this problem. The immune system normally recognizes the new gene as foreign and rejects the cells carrying it. The research utilized a newly uncovered network of genes regulated by molecules known as microRNAs. This natural function selectively obscured their therapeutic gene in immune system cells and protected it from discovery. Mice infected with the gene containing an immune-cell microRNA target sequence did not reject the gene.

In August scientists successfully treated metastatic melanoma in two patients using killer T cells genetically retargeted to attack the cancer cells.[74]

In November researchers reported on the use of VRX496, a gene-based immunotherapy for the treatment of HIV that uses a lentiviral vector to deliver an antisense gene against the HIV envelope. In a phase I clinical trial, five subjects with chronic HIV infection who had failed to respond to at least two antiretroviral regimens were treated. A single intravenous infusion of autologous CD4 T cells genetically modified with VRX496 was well tolerated. All patients had stable or decreased viral load; four of the five patients had stable or increased CD4 T cell counts. All five patients had stable or increased immune response to HIV antigens and other pathogens. This was the first evaluation of a lentiviral vector administered in a US human clinical trial.[75][76]

In May researchers announced the first gene therapy trial for inherited retinal disease. The first operation was carried out on a 23-year-old British male, Robert Johnson, in early 2007.[77]

Leber’s congenital amaurosis is an inherited blinding disease caused by mutations in the RPE65 gene. The results of a small clinical trial in children were published in April.[11] Delivery of recombinant adeno-associated virus (AAV) carrying RPE65 yielded positive results. In May two more groups reported positive results in independent clinical trials using gene therapy to treat the condition. In all three clinical trials, patients recovered functional vision without apparent side-effects.[11][12][13][14]

In September researchers were able to give trichromatic vision to squirrel monkeys.[78] In November 2009, researchers halted a fatal genetic disorder called adrenoleukodystrophy in two children using a lentivirus vector to deliver a functioning version of ABCD1, the gene that is mutated in the disorder.[79]

An April paper reported that gene therapy addressed achromatopsia (color blindness) in dogs by targeting cone photoreceptors. Cone function and day vision were restored for at least 33 months in two young specimens. The therapy was less efficient for older dogs.[80]

In September it was announced that an 18-year-old male patient in France with beta-thalassemia major had been successfully treated.[81] Beta-thalassemia major is an inherited blood disease in which beta haemoglobin is missing and patients are dependent on regular lifelong blood transfusions.[82] The technique used a lentiviral vector to transduce the human -globin gene into purified blood and marrow cells obtained from the patient in June 2007.[83] The patient’s haemoglobin levels were stable at 9 to 10 g/dL. About a third of the hemoglobin contained the form introduced by the viral vector and blood transfusions were not needed.[83][84] Further clinical trials were planned.[85] Bone marrow transplants are the only cure for thalassemia, but 75% of patients do not find a matching donor.[84]

Cancer immunogene therapy using modified antigene, antisense/triple helix approach was introduced in South America in 2010/11 in La Sabana University, Bogota (Ethical Committee 14 December 2010, no P-004-10). Considering the ethical aspect of gene diagnostic and gene therapy targeting IGF-I, the IGF-I expressing tumors i.e. lung and epidermis cancers were treated (Trojan et al. 2016).[86][87]

In 2007 and 2008, a man (Timothy Ray Brown) was cured of HIV by repeated hematopoietic stem cell transplantation (see also allogeneic stem cell transplantation, allogeneic bone marrow transplantation, allotransplantation) with double-delta-32 mutation which disables the CCR5 receptor. This cure was accepted by the medical community in 2011.[88] It required complete ablation of existing bone marrow, which is very debilitating.

In August two of three subjects of a pilot study were confirmed to have been cured from chronic lymphocytic leukemia (CLL). The therapy used genetically modified T cells to attack cells that expressed the CD19 protein to fight the disease.[20] In 2013, the researchers announced that 26 of 59 patients had achieved complete remission and the original patient had remained tumor-free.[89]

Human HGF plasmid DNA therapy of cardiomyocytes is being examined as a potential treatment for coronary artery disease as well as treatment for the damage that occurs to the heart after myocardial infarction.[90][91]

In 2011 Neovasculgen was registered in Russia as the first-in-class gene-therapy drug for treatment of peripheral artery disease, including critical limb ischemia; it delivers the gene encoding for VEGF.[92][26] Neovasculogen is a plasmid encoding the CMV promoter and the 165 amino acid form of VEGF.[93][94]

The FDA approved Phase 1 clinical trials on thalassemia major patients in the US for 10 participants in July.[95] The study was expected to continue until 2015.[85]

In July 2012, the European Medicines Agency recommended approval of a gene therapy treatment for the first time in either Europe or the United States. The treatment used Alipogene tiparvovec (Glybera) to compensate for lipoprotein lipase deficiency, which can cause severe pancreatitis.[96] The recommendation was endorsed by the European Commission in November 2012[10][27][97][98] and commercial rollout began in late 2014.[99] Alipogene tiparvovec was expected to cost around $1.6 million per treatment in 2012,[100] revised to $1 million in 2015,[101] making it the most expensive medicine in the world at the time.[102] As of 2016[update], only one person had been treated with drug.[103]

In December 2012, it was reported that 10 of 13 patients with multiple myeloma were in remission “or very close to it” three months after being injected with a treatment involving genetically engineered T cells to target proteins NY-ESO-1 and LAGE-1, which exist only on cancerous myeloma cells.[22]

In March researchers reported that three of five adult subjects who had acute lymphocytic leukemia (ALL) had been in remission for five months to two years after being treated with genetically modified T cells which attacked cells with CD19 genes on their surface, i.e. all B-cells, cancerous or not. The researchers believed that the patients’ immune systems would make normal T-cells and B-cells after a couple of months. They were also given bone marrow. One patient relapsed and died and one died of a blood clot unrelated to the disease.[21]

Following encouraging Phase 1 trials, in April, researchers announced they were starting Phase 2 clinical trials (called CUPID2 and SERCA-LVAD) on 250 patients[104] at several hospitals to combat heart disease. The therapy was designed to increase the levels of SERCA2, a protein in heart muscles, improving muscle function.[105] The FDA granted this a Breakthrough Therapy Designation to accelerate the trial and approval process.[106] In 2016 it was reported that no improvement was found from the CUPID 2 trial.[107]

In July researchers reported promising results for six children with two severe hereditary diseases had been treated with a partially deactivated lentivirus to replace a faulty gene and after 732 months. Three of the children had metachromatic leukodystrophy, which causes children to lose cognitive and motor skills.[108] The other children had Wiskott-Aldrich syndrome, which leaves them to open to infection, autoimmune diseases, and cancer.[109] Follow up trials with gene therapy on another six children with Wiskott-Aldrich syndrome were also reported as promising.[110][111]

In October researchers reported that two children born with adenosine deaminase severe combined immunodeficiency disease (ADA-SCID) had been treated with genetically engineered stem cells 18 months previously and that their immune systems were showing signs of full recovery. Another three children were making progress.[18] In 2014 a further 18 children with ADA-SCID were cured by gene therapy.[112] ADA-SCID children have no functioning immune system and are sometimes known as “bubble children.”[18]

Also in October researchers reported that they had treated six hemophilia sufferers in early 2011 using an adeno-associated virus. Over two years later all six were producing clotting factor.[18][113]

In January researchers reported that six choroideremia patients had been treated with adeno-associated virus with a copy of REP1. Over a six-month to two-year period all had improved their sight.[114][115] By 2016, 32 patients had been treated with positive results and researchers were hopeful the treatment would be long-lasting.[15] Choroideremia is an inherited genetic eye disease with no approved treatment, leading to loss of sight.

In March researchers reported that 12 HIV patients had been treated since 2009 in a trial with a genetically engineered virus with a rare mutation (CCR5 deficiency) known to protect against HIV with promising results.[116][117]

Clinical trials of gene therapy for sickle cell disease were started in 2014.[118][119] There is a need for high quality randomised controlled trials assessing the risks and benefits involved with gene therapy for people with sickle cell disease.[120]

In February LentiGlobin BB305, a gene therapy treatment undergoing clinical trials for treatment of beta thalassemia gained FDA “breakthrough” status after several patients were able to forgo the frequent blood transfusions usually required to treat the disease.[121]

In March researchers delivered a recombinant gene encoding a broadly neutralizing antibody into monkeys infected with simian HIV; the monkeys’ cells produced the antibody, which cleared them of HIV. The technique is named immunoprophylaxis by gene transfer (IGT). Animal tests for antibodies to ebola, malaria, influenza, and hepatitis were underway.[122][123]

In March, scientists, including an inventor of CRISPR, Jennifer Doudna, urged a worldwide moratorium on germline gene therapy, writing “scientists should avoid even attempting, in lax jurisdictions, germline genome modification for clinical application in humans” until the full implications “are discussed among scientific and governmental organizations”.[124][125][126][127]

In October, researchers announced that they had treated a baby girl, Layla Richards, with an experimental treatment using donor T-cells genetically engineered using TALEN to attack cancer cells. One year after the treatment she was still free of her cancer (a highly aggressive form of acute lymphoblastic leukaemia [ALL]).[128] Children with highly aggressive ALL normally have a very poor prognosis and Layla’s disease had been regarded as terminal before the treatment.[129]

In December, scientists of major world academies called for a moratorium on inheritable human genome edits, including those related to CRISPR-Cas9 technologies[130] but that basic research including embryo gene editing should continue.[131]

In April the Committee for Medicinal Products for Human Use of the European Medicines Agency endorsed a gene therapy treatment called Strimvelis[132][133] and the European Commission approved it in June.[134] This treats children born with adenosine deaminase deficiency and who have no functioning immune system. This was the second gene therapy treatment to be approved in Europe.[135]

In October, Chinese scientists reported they had started a trial to genetically modify T-cells from 10 adult patients with lung cancer and reinject the modified T-cells back into their bodies to attack the cancer cells. The T-cells had the PD-1 protein (which stops or slows the immune response) removed using CRISPR-Cas9.[136][137]

A 2016 Cochrane systematic review looking at data from four trials on topical cystic fibrosis transmembrane conductance regulator (CFTR) gene therapy does not support its clinical use as a mist inhaled into the lungs to treat cystic fibrosis patients with lung infections. One of the four trials did find weak evidence that liposome-based CFTR gene transfer therapy may lead to a small respiratory improvement for people with CF. This weak evidence is not enough to make a clinical recommendation for routine CFTR gene therapy.[138]

In February Kite Pharma announced results from a clinical trial of CAR-T cells in around a hundred people with advanced Non-Hodgkin lymphoma.[139]

In March, French scientists reported on clinical research of gene therapy to treat sickle-cell disease.[140]

In August, the FDA approved tisagenlecleucel for acute lymphoblastic leukemia.[141] Tisagenlecleucel is an adoptive cell transfer therapy for B-cell acute lymphoblastic leukemia; T cells from a person with cancer are removed, genetically engineered to make a specific T-cell receptor (a chimeric T cell receptor, or “CAR-T”) that reacts to the cancer, and are administered back to the person. The T cells are engineered to target a protein called CD19 that is common on B cells. This is the first form of gene therapy to be approved in the United States. In October, a similar therapy called axicabtagene ciloleucel was approved for non-Hodgkin lymphoma.[142]

In December the results of using an adeno-associated virus with blood clotting factor VIII to treat nine haemophilia A patients were published. Six of the seven patients on the high dose regime increased the level of the blood clotting VIII to normal levels. The low and medium dose regimes had no effect on the patient’s blood clotting levels.[143][144]

In December, the FDA approved Luxturna, the first in vivo gene therapy, for the treatment of blindness due to Leber’s congenital amaurosis.[145] The price of this treatment was 850,000 US dollars for both eyes.[146][147]

Speculated uses for gene therapy include:

Athletes might adopt gene therapy technologies to improve their performance.[148] Gene doping is not known to occur, but multiple gene therapies may have such effects. Kayser et al. argue that gene doping could level the playing field if all athletes receive equal access. Critics claim that any therapeutic intervention for non-therapeutic/enhancement purposes compromises the ethical foundations of medicine and sports.[149]

Genetic engineering could be used to cure diseases, but also to change physical appearance, metabolism, and even improve physical capabilities and mental faculties such as memory and intelligence. Ethical claims about germline engineering include beliefs that every fetus has a right to remain genetically unmodified, that parents hold the right to genetically modify their offspring, and that every child has the right to be born free of preventable diseases.[150][151][152] For parents, genetic engineering could be seen as another child enhancement technique to add to diet, exercise, education, training, cosmetics, and plastic surgery.[153][154] Another theorist claims that moral concerns limit but do not prohibit germline engineering.[155]

Possible regulatory schemes include a complete ban, provision to everyone, or professional self-regulation. The American Medical Associations Council on Ethical and Judicial Affairs stated that “genetic interventions to enhance traits should be considered permissible only in severely restricted situations: (1) clear and meaningful benefits to the fetus or child; (2) no trade-off with other characteristics or traits; and (3) equal access to the genetic technology, irrespective of income or other socioeconomic characteristics.”[156]

As early in the history of biotechnology as 1990, there have been scientists opposed to attempts to modify the human germline using these new tools,[157] and such concerns have continued as technology progressed.[158][159] With the advent of new techniques like CRISPR, in March 2015 a group of scientists urged a worldwide moratorium on clinical use of gene editing technologies to edit the human genome in a way that can be inherited.[124][125][126][127] In April 2015, researchers sparked controversy when they reported results of basic research to edit the DNA of non-viable human embryos using CRISPR.[160][161] A committee of the American National Academy of Sciences and National Academy of Medicine gave qualified support to human genome editing in 2017[162][163] once answers have been found to safety and efficiency problems “but only for serious conditions under stringent oversight.”[164]

Regulations covering genetic modification are part of general guidelines about human-involved biomedical research. There are no international treaties which are legally binding in this area, but there are recommendations for national laws from various bodies.

The Helsinki Declaration (Ethical Principles for Medical Research Involving Human Subjects) was amended by the World Medical Association’s General Assembly in 2008. This document provides principles physicians and researchers must consider when involving humans as research subjects. The Statement on Gene Therapy Research initiated by the Human Genome Organization (HUGO) in 2001 provides a legal baseline for all countries. HUGOs document emphasizes human freedom and adherence to human rights, and offers recommendations for somatic gene therapy, including the importance of recognizing public concerns about such research.[165]

No federal legislation lays out protocols or restrictions about human genetic engineering. This subject is governed by overlapping regulations from local and federal agencies, including the Department of Health and Human Services, the FDA and NIH’s Recombinant DNA Advisory Committee. Researchers seeking federal funds for an investigational new drug application, (commonly the case for somatic human genetic engineering,) must obey international and federal guidelines for the protection of human subjects.[166]

NIH serves as the main gene therapy regulator for federally funded research. Privately funded research is advised to follow these regulations. NIH provides funding for research that develops or enhances genetic engineering techniques and to evaluate the ethics and quality in current research. The NIH maintains a mandatory registry of human genetic engineering research protocols that includes all federally funded projects.

An NIH advisory committee published a set of guidelines on gene manipulation.[167] The guidelines discuss lab safety as well as human test subjects and various experimental types that involve genetic changes. Several sections specifically pertain to human genetic engineering, including Section III-C-1. This section describes required review processes and other aspects when seeking approval to begin clinical research involving genetic transfer into a human patient.[168] The protocol for a gene therapy clinical trial must be approved by the NIH’s Recombinant DNA Advisory Committee prior to any clinical trial beginning; this is different from any other kind of clinical trial.[167]

As with other kinds of drugs, the FDA regulates the quality and safety of gene therapy products and supervises how these products are used clinically. Therapeutic alteration of the human genome falls under the same regulatory requirements as any other medical treatment. Research involving human subjects, such as clinical trials, must be reviewed and approved by the FDA and an Institutional Review Board.[169][170]

Gene therapy is the basis for the plotline of the film I Am Legend[171] and the TV show Will Gene Therapy Change the Human Race?.[172] In 1994, gene therapy was a plot element in The Erlenmeyer Flask, The X-Files’ first-season finale. It is also used in Stargate as a means of allowing humans to use Ancient technology.[173]

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Gene therapy – Wikipedia

Gene therapy | definition of gene therapy by Medical …

Gene therapy is a rapidly growing field of medicine in which genes are introduced into the body to treat diseases. Genes control heredity and provide the basic biological code for determining a cell’s specific functions. Gene therapy seeks to provide genes that correct or supplant the disease-controlling functions of cells that are not, in essence, doing their job. Somatic gene therapy introduces therapeutic genes at the tissue or cellular level to treat a specific individual. Germ-line gene therapy inserts genes into reproductive cells or possibly into embryos to correct genetic defects that could be passed on to future generations. Initially conceived as an approach for treating inherited diseases, like cystic fibrosis and Huntington’s disease, the scope of potential gene therapies has grown to include treatments for cancers, arthritis, and infectious diseases. Although gene therapy testing in humans has advanced rapidly, many questions surround its use. For example, some scientists are concerned that the therapeutic genes themselves may cause disease. Others fear that germ-line gene therapy may be used to control human development in ways not connected with disease, like intelligence or appearance.The biological basis of gene therapy

Gene therapy has grown out of the science of genetics or how heredity works. Scientists know that life begins in a cell, the basic building block of all multicellular organisms. Humans, for instance, are made up of trillions of cells, each performing a specific function. Within the cell’s nucleus (the center part of a cell that regulates its chemical functions) are pairs of chromosomes. These threadlike structures are made up of a single molecule of DNA (deoxyribonucleic acid), which carries the blueprint of life in the form of codes, or genes, that determine inherited characteristics.

A DNA molecule looks like two ladders with one of the sides taken off both and then twisted around each other. The rungs of these ladders meet (resulting in a spiral staircase-like structure) and are called base pairs. Base pairs are made up of nitrogen molecules and arranged in specific sequences. Millions of these base pairs, or sequences, can make up a single gene, specifically defined as a segment of the chromosome and DNA that contains certain hereditary information. The gene, or combination of genes formed by these base pairs ultimately direct an organism’s growth and characteristics through the production of certain chemicals, primarily proteins, which carry out most of the body’s chemical functions and biological reactions.

Scientists have known how to manipulate a gene’s structure in the laboratory since the early 1970s through a process called gene splicing. The process involves removing a fragment of DNA containing the specific genetic sequence desired, then inserting it into the DNA of another gene. The resultant product is called recombinant DNA and the process is genetic engineering.

There are basically two types of gene therapy. Germ-line gene therapy introduces genes into reproductive cells (sperm and eggs) or someday possibly into embryos in hopes of correcting genetic abnormalities that could be passed on to future generations. Most of the current work in applying gene therapy, however, has been in the realm of somatic gene therapy. In this type of gene therapy, therapeutic genes are inserted into tissue or cells to produce a naturally occurring protein or substance that is lacking or not functioning correctly in an individual patient.

In both types of therapy, scientists need something to transport either the entire gene or a recombinant DNA to the cell’s nucleus, where the chromosomes and DNA reside. In essence, vectors are molecular delivery trucks. One of the first and most popular vectors developed were viruses because they invade cells as part of the natural infection process. Viruses have the potential to be excellent vectors because they have a specific relationship with the host in that they colonize certain cell types and tissues in specific organs. As a result, vectors are chosen according to their attraction to certain cells and areas of the body.

One of the first vectors used was retroviruses. Because these viruses are easily cloned (artificially reproduced) in the laboratory, scientists have studied them extensively and learned a great deal about their biological action. They also have learned how to remove the genetic information that governs viral replication, thus reducing the chances of infection.

Retroviruses work best in actively dividing cells, but cells in the body are relatively stable and do not divide often. As a result, these cells are used primarily for ex vivo (outside the body) manipulation. First, the cells are removed from the patient’s body, and the virus, or vector, carrying the gene is inserted into them. Next, the cells are placed into a nutrient culture where they grow and replicate. Once enough cells are gathered, they are returned to the body, usually by injection into the blood stream. Theoretically, as long as these cells survive, they will provide the desired therapy.

Scientists also have delved into nonviral vectors. These vectors rely on the natural biological process in which cells uptake (or gather) macromolecules. One approach is to use liposomes, globules of fat produced by the body and taken up by cells. Scientists also are investigating the introduction of raw recombinant DNA by injecting it into the bloodstream or placing it on microscopic beads of gold shot into the skin with a “gene-gun.” Another possible vector under development is based on dendrimer molecules. A class of polymers (naturally occurring or artificial substances that have a high molecular weight and formed by smaller molecules of the same or similar substances), is “constructed” in the laboratory by combining these smaller molecules. They have been used in manufacturing Styrofoam, polyethylene cartons, and Plexiglass. In the laboratory, dendrimers have shown the ability to transport genetic material into human cells. They also can be designed to form an affinity for particular cell membranes by attaching to certain sugars and protein groups.

In the early 1970s, scientists proposed “gene surgery” for treating inherited diseases caused by faulty genes. The idea was to take out the disease-causing gene and surgically implant a gene that functioned properly. Although sound in theory, scientists, then and now, lack the biological knowledge or technical expertise needed to perform such a precise surgery in the human body.

However, in 1983, a group of scientists from Baylor College of Medicine in Houston, Texas, proposed that gene therapy could one day be a viable approach for treating Lesch-Nyhan disease, a rare neurological disorder. The scientists conducted experiments in which an enzyme-producing gene (a specific type of protein) for correcting the disease was injected into a group of cells for replication. The scientists theorized the cells could then be injected into people with Lesch-Nyhan disease, thus correcting the genetic defect that caused the disease.

On September 14, 1990, a four-year old girl suffering from a genetic disorder that prevented her body from producing a crucial enzyme became the first person to undergo gene therapy in the United States. Because her body could not produce adenosine deaminase (ADA), she had a weakened immune system, making her extremely susceptible to severe, life-threatening infections. W. French Anderson and colleagues at the National Institutes of Health’s Clinical Center in Bethesda, Maryland, took white blood cells (which are crucial to proper immune system functioning) from the girl, inserted ADA producing genes into them, and then transfused the cells back into the patient. Although the young girl continued to show an increased ability to produce ADA, debate arose as to whether the improvement resulted from the gene therapy or from an additional drug treatment she received.

Nevertheless, a new era of gene therapy began as more and more scientists sought to conduct clinical trial (testing in humans) research in this area. In that same year, gene therapy was tested on patients suffering from melanoma (skin cancer). The goal was to help them produce antibodies (disease fighting substances in the immune system) to battle the cancer.

These experiments have spawned an ever growing number of attempts at gene therapies designed to perform a variety of functions in the body. For example, a gene therapy for cystic fibrosis aims to supply a gene that alters cells, enabling them to produce a specific protein to battle the disease. Another approach was used for brain cancer patients, in which the inserted gene was designed to make the cancer cells more likely to respond to drug treatment. Another gene therapy approach for patients suffering from artery blockage, which can lead to strokes, induces the growth of new blood vessels near clogged arteries, thus ensuring normal blood circulation.

The medical establishment’s contribution to transgenic research has been supported by increased government funding. In 1991, the U.S. government provided $58 million for gene therapy research, with increases in funding of $15-40 million dollars a year over the following four years. With fierce competition over the promise of societal benefit in addition to huge profits, large pharmaceutical corporations have moved to the forefront of transgenic research. In an effort to be first in developing new therapies, and armed with billions of dollars of research funds, such corporations are making impressive strides toward making gene therapy a viable reality in the treatment of once elusive diseases.

The potential scope of gene therapy is enormous. More than 4,200 diseases have been identified as resulting directly from abnormal genes, and countless others that may be partially influenced by a person’s genetic makeup. Initial research has concentrated on developing gene therapies for diseases whose genetic origins have been established and for other diseases that can be cured or improved by substances genes produce.

The following are examples of potential gene therapies. People suffering from cystic fibrosis lack a gene needed to produce a salt-regulating protein. This protein regulates the flow of chloride into epithelial cells, (the cells that line the inner and outer skin layers) that cover the air passages of the nose and lungs. Without this regulation, patients with cystic fibrosis build up a thick mucus that makes them prone to lung infections. A gene therapy technique to correct this abnormality might employ an adenovirus to transfer a normal copy of what scientists call the cystic fibrosis transmembrane conductance regulator, or CTRF, gene. The gene is introduced into the patient by spraying it into the nose or lungs. Researchers announced in 2004 that they had, for the first time, treated a dominant neurogenerative disease called Spinocerebella ataxia type 1, with gene therapy. This could lead to treating similar diseases such as Huntingtons disease. They also announced a single intravenous injection could deliver therapy to all muscles, perhaps providing hope to people with muscular dystrophy.

Although great strides have been made in gene therapy in a relatively short time, its potential usefulness has been limited by lack of scientific data concerning the multitude of functions that genes control in the human body. For instance, it is now known that the vast majority of genetic material does not store information for the creation of proteins, but rather is involved in the control and regulation of gene expression, and is, thus, much more difficult to interpret. Even so, each individual cell in the body carries thousands of genes coding for proteins, with some estimates as high as 150,000 genes. For gene therapy to advance to its full potential, scientists must discover the biological role of each of these individual genes and where the base pairs that make them up are located on DNA.

To address this issue, the National Institutes of Health initiated the Human Genome Project in 1990. Led by James D. Watson (one of the co-discoverers of the chemical makeup of DNA) the project’s 15-year goal is to map the entire human genome (a combination of the words gene and chromosomes). A genome map would clearly identify the location of all genes as well as the more than three billion base pairs that make them up. With a precise knowledge of gene locations and functions, scientists may one day be able to conquer or control diseases that have plagued humanity for centuries.

Gene therapy seems elegantly simple in its concept: supply the human body with a gene that can correct a biological malfunction that causes a disease. However, there are many obstacles and some distinct questions concerning the viability of gene therapy. For example, viral vectors must be carefully controlled lest they infect the patient with a viral disease. Some vectors, like retroviruses, also can enter cells functioning properly and interfere with the natural biological processes, possibly leading to other diseases. Other viral vectors, like the adenoviruses, often are recognized and destroyed by the immune system so their therapeutic effects are short-lived. Maintaining gene expression so it performs its role properly after vector delivery is difficult. As a result, some therapies need to be repeated often to provide long-lasting benefits.

One of the most pressing issues, however, is gene regulation. Genes work in concert to regulate their functioning. In other words, several genes may play a part in turning other genes on and off. For example, certain genes work together to stimulate cell division and growth, but if these are not regulated, the inserted genes could cause tumor formation and cancer. Another difficulty is learning how to make the gene go into action only when needed. For the best and safest therapeutic effort, a specific gene should turn on, for example, when certain levels of a protein or enzyme are low and must be replaced. But the gene also should remain dormant when not needed to ensure it doesn’t oversupply a substance and disturb the body’s delicate chemical makeup.

One approach to gene regulation is to attach other genes that detect certain biological activities and then react as a type of automatic off-and-on switch that regulates the activity of the other genes according to biological cues. Although still in the rudimentary stages, researchers are making headway in inhibiting some gene functioning by using a synthetic DNA to block gene transcriptions (the copying of genetic information). This approach may have implications for gene therapy.

While gene therapy holds promise as a revolutionary approach to treating disease, ethical concerns over its use and ramifications have been expressed by scientists and lay people alike. For example, since much needs to be learned about how these genes actually work and their long-term effect, is it ethical to test these therapies on humans, where they could have a disastrous result? As with most clinical trials concerning new therapies, including many drugs, the patients participating in these studies usually have not responded to more established therapies and often are so ill the novel therapy is their only hope for long-term survival.

Another questionable outgrowth of gene therapy is that scientists could possibly manipulate genes to genetically control traits in human offspring that are not health related. For example, perhaps a gene could be inserted to ensure that a child would not be bald, a seemingly harmless goal. However, what if genetic manipulation was used to alter skin color, prevent homosexuality, or ensure good looks? If a gene is found that can enhance intelligence of children who are not yet born, will everyone in society, the rich and the poor, have access to the technology or will it be so expensive only the elite can afford it?

The Human Genome Project, which plays such an integral role for the future of gene therapy, also has social repercussions. If individual genetic codes can be determined, will such information be used against people? For example, will someone more susceptible to a disease have to pay higher insurance premiums or be denied health insurance altogether? Will employers discriminate between two potential employees, one with a “healthy” genome and the other with genetic abnormalities?

Some of these concerns can be traced back to the eugenics movement popular in the first half of the twentieth century. This genetic “philosophy” was a societal movement that encouraged people with “positive” traits to reproduce while those with less desirable traits were sanctioned from having children. Eugenics was used to pass strict immigration laws in the United States, barring less suitable people from entering the country lest they reduce the quality of the country’s collective gene pool. Probably the most notorious example of eugenics in action was the rise of Nazism in Germany, which resulted in the Eugenic Sterilization Law of 1933. The law required sterilization for those suffering from certain disabilities and even for some who were simply deemed “ugly.” To ensure that this novel science is not abused, many governments have established organizations specifically for overseeing the development of gene therapy. In the United States, the Food and Drug Administration (FDA) and the National Institutes of Health require scientists to take a precise series of steps and meet stringent requirements before proceeding with clinical trials. As of mid-2004, more than 300 companies were carrying out gene medicine developments and 500 clinical trials were underway. How to deliver the therapy is the key to unlocking many of the researchers discoveries.

In fact, gene therapy has been immersed in more controversy and surrounded by more scrutiny in both the health and ethical arena than most other technologies (except, perhaps, for cloning) that promise to substantially change society. Despite the health and ethical questions surrounding gene therapy, the field will continue to grow and is likely to change medicine faster than any previous medical advancement.

Abella, Harold. “Gene Therapy May Save Limbs.” Diagnostic Imaging (May 1, 2003): 16.

Christensen R. “Cutaneous Gene TherapyAn Update.” Histochemical Cell Biology (January 2001): 73-82.

“Gene Therapy Important Part of Cancer Research.” Cancer Gene Therapy Week (June 30, 2003): 12.

“Initial Sequencing and Analysis of the Human Genome.” Nature (February 15, 2001): 860-921.

Kingsman, Alan. “Gene Therapy Moves On.” SCRIP World Pharmaceutical News (July 7, 2004): 19:ndash;21.

Nevin, Norman. “What Has Happened to Gene Therapy?” European Journal of Pediatrics (2000): S240-S242.

“New DNA Vaccine Targets Proteins Expressed in Cervical Cancer Cells.” Gene Therapy Weekly (September 9, 2004): 14.

“New Research on the Progress of Gene Therapy Presented at Meeting.” Obesity, Fitness & Wellness Week (July 3, 2004): 405.

Pekkanen, John. “Genetics: Medicine’s Amazing Leap.” Readers Digest (September 1991): 23-32.

Silverman, Jennifer, and Steve Perlstein. “Genome Project Completed.” Family Practice News (May 15, 2003): 50-51.

“Study Highlights Potential Danger of Gene Therapy.” Drug Week (June 20, 2003): 495.

“Study May Help Scientists Develop Safer Mthods for Gene Therapy.” AIDS Weekly (June 30, 2003): 32.

Trabis, J. “With Gene Therapy, Ears Grow New Sensory Cells.” Science News (June 7, 2003): 355.

National Human Genome Research Institute. The National Institutes of Health. 9000 Rockville Pike, Bethesda, MD 20892. (301) 496-2433. http://www.nhgri.nih.gov.

Online Mendelian Inheritance in Man. Online genetic testing information sponsored by National Center for Biotechnology Information. http://www.ncbi.nlm.nih.gov/Omim/.

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Gene Therapy – Learn.Genetics

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Genetic Science Learning Center. (2012, December 1) Gene Therapy.Retrieved October 10, 2018, from https://learn.genetics.utah.edu/content/genetherapy/

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Gene Therapy [Internet]. Salt Lake City (UT): Genetic Science Learning Center; 2012[cited 2018 Oct 10] Available from https://learn.genetics.utah.edu/content/genetherapy/

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Genetic Science Learning Center. “Gene Therapy.” Learn.Genetics.December 1, 2012. Accessed October 10, 2018. https://learn.genetics.utah.edu/content/genetherapy/.

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How Does Gene Therapy Work?

Scientists have promised that gene therapy will be the next big leap for medicine. It’s a term that’s tossed about regularly, but what is it exactly? Trace shows us how scientists can change your very genetic code.

Read More:

How does gene therapy work?http://ghr.nlm.nih.gov/handbook/thera…”Gene therapy is designed to introduce genetic material into cells to compensate for abnormal genes or to make a beneficial protein. If a mutated gene causes a necessary protein to be faulty or missing, gene therapy may be able to introduce a normal copy of the gene to restore the function of the protein.”

Gene therapy trial ‘cures children’http://www.bbc.co.uk/news/health-2326…”A disease which robs children of the ability to walk and talk has been cured by pioneering gene therapy to correct errors in their DNA, say doctors.”

Gene therapy cures diabetic dogshttp://www.newscientist.com/article/d…”Five diabetic beagles no longer needed insulin injections after being given two extra genes, with two of them still alive more than four years later.”

Gene Therapy for Cancer: Questions and Answershttp://www.cancer.gov/cancertopics/fa…”Gene therapy is an experimental treatment that involves introducing genetic material into a person’s cells to fight or prevent disease.”

How does gene therapy work?http://www.scientificamerican.com/art…”Gene therapy is the addition of new genes to a patient’s cells to replace missing or malfunctioning genes. Researchers typically do this using a virus to carry the genetic cargo into cells, because that’s what viruses evolved to do with their own genetic material.”

Gene therapy cures leukaemia in eight dayshttp://www.newscientist.com/article/m…eight-days.htmlWITHIN just eight days of starting a novel gene therapy, David Aponte’s “incurable” leukaemia had vanished. For four other patients, the same happened within eight weeks, although one later died from a blood clot unrelated to the treatment, and another after relapsing.

Cell Therapy Shows Promise for Acute Type of Leukemiahttp://www.nytimes.com/2013/03/21/hea…”A treatment that genetically alters a patient’s own immune cells to fight cancer has, for the first time, produced remissions in adults with an acute leukemia that is usually lethal, researchers are reporting.”

Watch More:Tricking the Immune Systemhttp://www.youtube.com/watch?v=Kr_HRl…Babies with 3 Parents?!http://www.youtube.com/watch?v=jQxsW_…Pick Your Poison: Cyanidehttp://www.youtube.com/watch?v=JDBrdE…____________________

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How Does Gene Therapy Work?

Gene Therapy for Pediatric Diseases | DNA Therapy – Dana …

Gene therapy delivers DNAinto a patients cells to replace faulty or missing genes or adds new genes in an attempt to cure diseases or to make changes so the body is better able tofight off disease. The DNA for a gene or genes is carried into a patientscells by a delivery vehicle called a vector, typically a specially engineeredvirus. The vector then inserts the gene(s) into the cells’ DNA.

Although gene therapy is relativelynew and often still considered experimental, it can provide a cure for life-threateningdiseases that dont respond well to other therapies (includingimmunodeficiencies, metabolic disorders, and relapsed cancers) and for acuteconditions that currently rely on complex and expensive life-long medicationand management (such as sickle cell disease and hemophilia).

CAR T-Cell Therapy for Relapsed Acute Lymphoblastic Leukemia (ALL)

Dana-Farber/Boston Childrens is a certified treatment center for providing the recently-FDA-approved CAR T-cell therapy called KYMRIAH for relapsed B-cell acute lymphoblastic leukemia (ALL). This promising new treatment entails genetic engineering of the patients own T-cells to increase targeting of a specific leukemia protein and then accelerate killing of the target. After modification, they are returned to the patient via IV where they can immediately begin destroying circulating cancer cells.

For more information about CAR T-cell therapy, contact our gene therapy program.

Our Gene Therapy Clinical Trials

Learn more about our gene therapy clinical trials.

Dana-Farber/BostonChildrens has one the most extensive and long-running pediatric gene therapyprograms in the world. Since 2010, wehave treated 36 patients from 11 countries through eight gene therapy clinicaltrials.

Why choose Dana-Farber/BostonChildrens:

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Gene Therapy for Pediatric Diseases | DNA Therapy – Dana …


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