China Is Trying to Scrub Bikinis and Smoking From the Internet

A new story reveals how Chinese live-streaming company Inke uses a combination of human moderators and AI to facilitate government censorship.

Cleaning Cyberspace

On Monday, the South China Morning Post published a story about the content moderation operations at Inke, one of China’s largest live-streaming companies.

The piece offers a rare glimpse at how China’s private sector helps facilitate government censorship. In some cases, that means flagging streams of people smoking or wearing bikinis — content that would likely seem fairly innocuous to an American audience — but in others, it means preventing internet viewers from seeing streams of people committing acts of terrorism or violence.

That’s the same kind of content multinational corporations such as Facebook have had trouble moderating — raising questions about what these Chinese companies have figured out that American ones haven’t.

Evolving Censorship

Inke tasks a team of 1,200 moderators with policing the streams of its 25 million users, according to SCMP.

The moderators watch streams 10- to 15-seconds before they actually go live, and in that time, they’re expected to catch anything “that is against the law and regulations, against mainstream values, and against the company’s values,” Zhi Heng, Inke’s content safety team leader, told the SCMP.

Inke defers to guidelines published by the China Association of Performing Arts to know what content falls under that umbrella, and according to the SCMP story, it ranges from politically sensitive speech and violence to people smoking or wearing bikinis.

The document is updated weekly, however, meaning content that might be acceptable one week could be censored the next, or vice versa.

To make this massive task of censoring content a little more manageable on its human moderators, Inke also employs algorithms and recognition software capable of filtering content into different risk categories.

The company sometimes dedicates just one human reviewer to watching streams considered “low-risk,” such as cooking shows, according to SCMP, while higher-risk streams receive closer scrutiny.

Learning Opportunity

The idea of censoring streams of people smoking cigarettes or wearing bikinis might seem ridiculous to a Western audience.

However, if Inke’s combination of human and AI moderators is effective at flagging the content deemed objectionable in China, it’s worth considering what it’s doing that others, such as Facebook, aren’t. Are Inke’s algorithms better in some discernible way? Has it stumbled upon the optimum human moderator-to-user ratio?

You might not agree with the content China is censoring, but content moderation isn’t by default objectionable — even Facebook’s own execs believe the company should have prevented the horrific livestream of the Christchurch shooting from reaching its audience, for example.

So perhaps there’s something Facebook and others could learn from how Inke is managing the job of filtering out undesirable online content, even if we don’t agree with China’s definition of undesirable.

READ MORE: No smoking, no tattoos, no bikinis: inside China’s war to ‘clean up’ the internet [South China Morning Post]

More on censorship: China Is Censoring “Genetically Edited Babies” on Social Media

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China Is Trying to Scrub Bikinis and Smoking From the Internet

Undersea Robots Are Helping Save the Great Barrier Reef

Australian scientists are preparing to deliver millions of coral larvae to the Great Barrier Reef using an autonomous drone called

RoboStork

A team of Australian scientists built an underwater robot that can deliver larval coral to the Great Barrier reef, where they hope it will help restore the reef to some of its former glory, before it was ravaged by climate change.

The delivery drone, LarvalBot, is a more hospitable version of the underwater drone that has previously been used to hunt and kill off the coral’s predators — yet another experiment in using robotics to protect and help recover the world’s coral reefs.

Fertilizing The Lawn

The scientists behind the project consider their work similar to fertilizing a lawn, according to Particle. Except instead of grass, it’s working on a beautiful and complex underwater ecosystem.

In order to re-seed the coral reef with larvae, scientists first need to gather that seed in the first place. Back in November, the researchers gathered millions of coral sperm and egg cells for what they called at the time “IVF for coral.”

Planning In Advance

LarvalBot made its first delivery back in December. Now the researchers are planning a second expedition to coincide with the reef’s natural mass spawning period, which will happen in October into November.

When that happens, LarvalBot will dive down, dropping millions of larvae that the researchers hope will be able to take root as brand new coral.

READ MORE: ROBOTS TO THE RESCUE OF THE GREAT BARRIER REEF [Particle]

More on the coral reef: To Protect Endangered Coral Reefs, Researchers Need Legal Recourse

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Undersea Robots Are Helping Save the Great Barrier Reef

Walmart Is Rolling Out Floor-Cleaning Robots in 1,500 Stores

Walmart is sending autonomous custodial robots to 1,500 stores in a play to cut down on the tasks human employees have to face.

Clean Many Robots

Walmart is about to bring worker robots to a third of its stores.

Of the corporation’s 4,600 U.S. locations, 1,500 are about to start using floor-cleaning custodial robots and 300 will use the bots to spot empty shelves, according to The Wall Street Journal. It’s a move that could save human employees a lot of time, but also one that signals that Walmart considers sees human employees and their salaries as circumventable expenses.

Time To Pivot

“With automation, we are able to take away some of the tasks that associates don’t enjoy doing,” Mark Propes, a Walmart operations director, told the WSJ. “At the same time, we continue to open up new jobs in other things in the store.”

Those other jobs are likely related to e-commerce, WSJ reports, as Walmart plans to pivot to more online sales in an attempt to challenge Amazon.

READ MORE: Walmart Is Rolling Out the Robots [The Wall Street Journal]

More on Walmart: Walmart Is About to Deploy Hundreds of Robot Janitors

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Walmart Is Rolling Out Floor-Cleaning Robots in 1,500 Stores

Zapping Elderly People’s Brains Supercharges Their Working Memory

Electrically stimulating the brains of people in their 60s and 70s allowed them to perform as well on working memory tasks as 20-somethings.

Memory Games

Stimulating the brains of elderly people with electrical currents allowed them to perform just as well on a memory test as people in their 20s — a sign that researchers may have found a noninvasive way to turn back the hands of time when it comes to human memory.

“It’s opening up a whole new avenue of potential research and treatment options,” researcher Rob Reinhart said in a press release regarding the study, “and we’re super excited about it.”

All Ages

In a study published in the journal Nature Neuroscience on Monday, researchers from Boston University detail how they asked a group of 20-somethings and a group of people in their 60s and 70s to complete a task designed to test their working memory, which is the part of our short-term memory that we use for reasoning and decision-making.

Working memory typically begins declining around the time we hit 30 years old, so as expected, the people in their 20s outperformed the older group on the memory task.

Remembrall

However, after the members of the older group received 25 minutes of mild stimulation via scalp electrodes, they performed just as well as the younger participants — and the memory boost still hadn’t subsided by the time the experiment ended 50 minutes later.

According to the researchers, the benefits of this noninvasive treatment could extend beyond those whose working memory has started to succumb to age, too. They found that stimulating the brains of the younger people who performed poorly on the task boosted their memories as well.

READ MORE: As Memories Fade, Can We Supercharge Them Back to Life? [Boston University]

More on memory: Can a Brain Zap Really Boost Your Memory?

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Zapping Elderly People’s Brains Supercharges Their Working Memory

The Israeli Moon Lander Is About to Touch Down

SpaceIL's Moon lander, Beresheet, is expected to touch down on the lunar surface on Thursday, landing Israeli a place in the history books.

Lunar Lander

If all goes according to plan, Israel will earn a place in history on Thursday as the fourth nation ever to land a spacecraft on the Moon — and unlike any craft that came before it, this Moon lander was privately funded.

Beresheet is the work of SpaceIL, a nonprofit Israeli space company. On Feb. 21, the company launched its $100 million spacecraft on a journey to the Moon aboard a SpaceX Falcon 9 rocket, and on April 4, it settled into the Moon’s orbit.

The next step in the mission is for Beresheet to attempt to land on the surface of the Moon sometime between 3 and 4 p.m. ET on Thursday.

Watch Along

Beresheet’s target landing site is in the northeastern part of Mare Serenitatis, also known as the Sea of Serenity.

“On the basis of our experience with Apollo, the Serenitatis sites favor both landing safety and scientific reward,” SpaceIL team member Jim Head said in a press release.

SpaceIL and Israel Aerospace Industries, the company that built Beresheet, will live-stream Thursday’s touch-down attempt, so the world will have a chance to watch along as Israel tries to land itself a spot in the history books.

READ MORE: Israel’s Beresheet space probe prepares for historic moon landing [NBC News]

More on Beresheet: Israel’s Moon Lander Just Got Photobombed by the Earth

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The Israeli Moon Lander Is About to Touch Down

Some People Are Exceptionally Good at Predicting the Future

Some people are adept at forecasting, predicting the likelihood of future events, and a new contest aims to suss them out.

Super-Forecasters

Some people have a knack for accurately predicting the likelihood of future events. You might even be one of these “super-forecasters” and not know it — but now there’s an easy way to find out.

BBC Future has teamed up with UK-based charity Nesta and forecasting services organization Good Judgement on the “You Predict the Future” challenge. The purpose is to study how individuals and teams predict the likelihood of certain events, ranging from the technological to the geopolitical.

All Winners

Anyone interested in testing their own forecasting skills can sign up for the challenge to answer a series of multiple-choice questions and assign a percentage to how likely each answer is to come true.

“When you’re part of the challenge, you’ll get feedback on how accurate your forecasts are,” Kathy Peach, who leads Nesta’s Centre for Collective Intelligence Design, told BBC Future. “You’ll be able to see how well you do compared to other forecasters. And there’s a leader board, which shows who the best performing forecasters are.”

Collective Intelligence

You’ll also be helping advance research on collective intelligence, which focuses on the intellectual abilities of groups of people acting as one.

Additionally, as Peach told BBC Future, “New research shows that forecasting increases open-mindedness, the ability to consider alternative scenarios, and reduces political polarisation,”  — meaning even if you don’t find out you’re a “super-forecaster,” you might just end up a better person after making your predictions.

READ MORE: Could you be a super-forecaster? [BBC Future]

More on forecasting: Forecasting the Future: Can the Hive Mind Let Us Predict the Future?

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Some People Are Exceptionally Good at Predicting the Future

Scientists Say New Quantum Material Could “‘Download’ Your Brain”

A new type of quantum material can directly measure neural activity and translate it into electrical signals for a computer.

Computer Brain

Scientists say they’ve developed a new “quantum material” that could one day transfer information directly from human brains to a computer.

The research is in early stages, but it invokes ideas like uploading brains to the cloud or hooking people up to a computer to track deep health metrics — concepts that until now existed solely in science fiction.

Quantum Interface

The new quantum material, described in research published Wednesday in the journal Nature Communications, is a “nickelate lattice” that the scientists say could directly translate the brain’s electrochemical signals into electrical activity that could be interpreted by a computer.

“We can confidently say that this material is a potential pathway to building a computing device that would store and transfer memories,” Purdue University engineer Shriram Ramanathan told ScienceBlog.

Running Diagnostics

Right now, the new material can only detect the activity of some neurotransmitters — so we can’t yet upload a whole brain or anything like that. But if the tech progresses, the researchers hypothesize that it could be used to detect neurological diseases, or perhaps even store memories.

“Imagine putting an electronic device in the brain, so that when natural brain functions start deteriorating, a person could still retrieve memories from that device,” Ramanathan said.

READ MORE: New Quantum Material Could Warn Of Neurological Disease [ScienceBlog]

More on brain-computer interface: This Neural Implant Accesses Your Brain Through the Jugular Vein

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Scientists Say New Quantum Material Could “‘Download’ Your Brain”

Scientists Find a New Way to Kickstart Stable Fusion Reactions

A new technique for nuclear fusion can generate plasma without requiring as much space-consuming equipment within a reactor.

Warm Fusion

Scientists from the Princeton Plasma Physics Laboratory say that they’ve found a new way to start up nuclear fusion reactions.

The new technique, described in research published last month in the journal Physics of Plasmas, provides an alternate means for reactors to convert gas into the superhot plasma that gets fusion reactions going with less equipment taking up valuable lab space — another step in the long road to practical fusion power.

Out With The Old

Right in the center of a tokamak, a common type of experimental nuclear fusion reactor, there’s a large central magnet that helps generate plasma. The new technique, called “transient coaxial helical injection,” does away with the magnet but still generates a stable reaction, freeing up the space taken up by the magnet for other equipment.

“The good news from this study,” Max Planck Institute researcher Kenneth Hammond said in a press release, “is that the projections for startup in large-scale devices look promising.”

READ MORE: Ready, set, go: Scientists evaluate novel technique for firing up fusion-reaction fuel [Princeton Plasma Physics Laboratory newsroom via ScienceDaily]

More on nuclear fusion: Scientists Found a New Way to Make Fusion Reactors More Efficient

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Scientists Find a New Way to Kickstart Stable Fusion Reactions

Infertile Couple Gives Birth to “Three-Parent Baby”

A Greek couple just gave birth to a three-parent baby, the first conceived as part of a clinical trial to treat infertility.

Happy Birthday

On Tuesday, a couple gave birth to what researchers are calling a “three-parent baby” — giving new hope to infertile couples across the globe.

After four cycles of in vitro fertilization failed to result in a pregnancy, the Greek couple enrolled in a clinical trial for mitochondrial replacement therapy (MRT) — meaning doctors placed the nucleus from the mother’s egg into a donor egg that had its nucleus removed. Then they fertilized the egg with sperm from the father and implanted it into the mother.

Due to this procedure, the six-pound baby boy has DNA from both his mother and father, as well as a tiny bit from the woman who donated the egg.

Greek Life

The Greek baby wasn’t the first “three-parent baby” born after his parents underwent MRT — that honor goes to the offspring of a Jordanian woman who gave birth in 2016.

However, in her case and others that followed it, doctors used the technique to prevent a baby from inheriting a parent’s genetic defect. This marked the first time a couple used MRT as part of a clinical trial to treat infertility.

“Our excellent collaboration and this exceptional result will help countless women to realise their dream of becoming mothers with their own genetic material,” Nuno Costa-Borges, co-founder of Embryotools, one of the companies behind the trial, said in a statement.

READ MORE: Baby with DNA from three people born in Greece [The Guardian]

More on three-parent babies: An Infertile Couple Is Now Pregnant With a “Three-Parent Baby”

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Infertile Couple Gives Birth to “Three-Parent Baby”

MIT Prof: If We Live in a Simulation, Are We Players or NPCs?

An MIT scientist asks whether we're protagonists in a simulated reality or so-called NPCs who exist to round out a player character's experience. 

Simulation Hypothesis

Futurism readers may recognize Rizwan Virk as the MIT researcher touting a new book arguing that we’re likely living in a game-like computer simulation.

Now, in new interview with Vox, Virk goes even further — by probing whether we’re protagonists in the simulation or so-called “non-player characters” who are presumably included to round out a player character’s experience.

Great Simulation

Virk speculated about whether we’re players or side characters when Vox writer Sean Illing asked a question likely pondered by anyone who’s seen “The Matrix”: If you were living in a simulation, would you actually want to know?

“Probably the most important question related to this is whether we are NPCs (non-player characters) or PCs (player characters) in the video game,” Virk told Vox. “If we are PCs, then that means we are just playing a character inside the video game of life, which I call the Great Simulation.”

More Frightening

It’s a line of inquiry that cuts to the core of the simulation hypothesis: If the universe is essentially a video game, who built it — and why?

“The question is, are all of us NPCs in a simulation, and what is the purpose of that simulation?” Virk asked. “A knowledge of the fact that we’re in a simulation, and the goals of the simulation and the goals of our character, I think, would still be interesting to many people.”

READ MORE: Are we living in a computer simulation? I don’t know. Probably. [Vox]

More on the simulation hypothesis: Famous Hacker Thinks We’re Living in Simulation, Wants to Escape

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MIT Prof: If We Live in a Simulation, Are We Players or NPCs?

Here’s How Big the M87 Black Hole Is Compared to the Earth

The black hole that scientists imaged is a stellar giant. It would take millions of Earths lined up side-by-side to span its length.

Pale Black Dot

On Wednesday, a team of scientists from around the world released the first ever directly-observed image of the event horizon of a black hole.

The black hole, M87*, is found within the constellation Virgo — and as the webcomic XKCD illustrated, it’s as big as our entire solar system.

Stellar Giant

The gigantic black hole, not counting the giant rings of trapped light orbiting it, is about 23.6 billion miles (38 billion kilometers) across, according to Science News.

Meanwhile, the Earth is just 7,917 miles in diameter — meaning our planet wouldn’t even be a drop in the bucket of the giant, black void. Based Futurism’s calculations, it would take just over 2.98 million Earths lined up in a row to span the length of M87*. For a sense of scale, that’s about how many adult giraffes it would take to span the diameter of Earth.

Paging Pluto

Our entire solar system is just about 2.27 billion miles wide, meaning we could just barely fit the whole thing into the newly-imaged black hole’s event horizon.

Thankfully, M87* is about 55 million light years away — so while we could readily fit inside its gaping maw, we’re way too far to get sucked in.

READ MORE: Revealed: a black hole the size of the solar system [Cosmos]

More on M87*: Scientists: Next Black Whole Image Will Be Way Clearer

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Here’s How Big the M87 Black Hole Is Compared to the Earth

Amazon Workers Listen to Your Alexa Conversations, Then Mock Them

A new Bloomberg piece shared the experiences of Amazon workers tasked with listening to Alexa recordings, and what they hear isn't always mundane.

I Hear You

Amazon pays thousands of workers across the globe to review audio picked up by its Echo speakers — and their behavior raises serious concerns about both privacy and safety.

Bloomberg recently spoke with seven people who participated in Amazon’s audio review process. Each worker was tasked with listening to, transcribing, and annotating voice recordings with the goal of improving the ability of Amazon’s Alexa smart assistant to understand and respond to human speech.

But sometimes, according to Bloomberg, they share private recordings in a disrespectful way.

“I think we’ve been conditioned to the [assumption] that these machines are just doing magic machine learning” University of Michigan professor Florian Schaub told Bloomberg. “But the fact is there is still manual processing involved.”

Listen to This

The job is usually boring, according to Bloomberg’s sources. But if they heard something out of the ordinary, they said, sometimes they’d share the Alexa recordings with other workers via internal chat rooms.

Occasionally, it was just because they found the audio amusing — a person singing off-key, for example — but other times, the sharing was “a way of relieving stress” after hearing something disturbing, such as when two of Bloomberg’s sources heard what sounded like a sexual assault.

When they asked Amazon how to handle cases like the latter, the workers said they were told “it wasn’t Amazon’s job to interfere.” Amazon, meanwhile, said it had procedures in place for when workers hear something “distressing” in Alexa recordings.

READ MORE: Amazon Workers Are Listening to What You Tell Alexa [Bloomberg]

More on Echo: Thanks, Amazon! Echo Recorded and Sent Audio to Random Contacts Without Warning

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Amazon Workers Listen to Your Alexa Conversations, Then Mock Them

NASA Is Funding the Development of 18 Bizarre New Projects

Through the NASA Innovative Advanced Concepts (NIAC) program, NASA funds projects that go

Nurturing the Bizarre

NASA isn’t afraid to take a chance on the weird. In fact, it has a program designed for that specific purpose, called NASA Innovative Advanced Concepts (NIAC) — and on Wednesday, the agency announced 18 bizarre new projects receiving funding through the program.

“Our NIAC program nurtures visionary ideas that could transform future NASA missions by investing in revolutionary technologies,” NASA exec Jim Reuter said in a press release. “We look to America’s innovators to help us push the boundaries of space exploration with new technology.”

Sci-Fi to Sci-Fact

The 18 newly funded projects are divided into two groups: Phase I and Phase II.

The 12 recipients of the Phase I awards will each receive approximately $125,000 to fund nine month’s worth of feasibility studies for their concepts. These include a project to beam power through Venus’ atmosphere to support long-term missions, a spacesuit with self-healing skin, and floating microprobes inspired by spiders.

The six Phase II recipients, meanwhile, will each receive up to $500,000 to support two-year studies dedicated to fine-tuning their concepts and investigating potential ways to implement the technologies, which include a flexible telescope, a neutrino detector, and materials for solar surfing.

“NIAC is about going to the edge of science fiction, but not over,” Jason Derleth, NIAC program executive, said in the press release. “We are supporting high impact technology concepts that could change how we explore within the solar system and beyond.”

READ MORE: NASA Invests in Potentially Revolutionary Tech Concepts [Jet Propulsion Laboratory]

More on bizarre NASA plans: New NASA Plan for Mars Is Moderately-Terrifying-Sounding, Also, Completely-Awesome: Robotic. Bees.

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NASA Is Funding the Development of 18 Bizarre New Projects

Report: Tesla Doc Is Playing Down Injuries to Block Workers’ Comp

Former Tesla and clinic employees share how doctors blocked workers' compensation claims and put injured people back to work to avoid payouts.

Here’s A Band-Aid

Tesla’s on-site clinic, Access Omnicare, has allegedly been downplaying workers’ injuries to keep the electric automaker off the hook for workers’ compensation.

Several former Tesla employees, all of whom got hurt on the job, and former employees of Access Omnicare, told Reveal News that the clinic was minimizing worker injuries so that the automaker wouldn’t have to pay workers’ comp — suggesting that the barely-profitable car company is willing to do whatever it takes to stay out of the red and avoid negative press.

Back To Work

Reveal, which is a project by the Center for Investigative Reporting, described cases in which employees suffered electrocution, broken bones, and mold-related rashes while working in a Tesla factory — only for Omnicare to deny that the injuries warranted time off work.

The clinic’s top doctor “wanted to make certain that we were doing what Tesla wanted so badly,” former Omnicare operations manager Yvette Bonnet told Reveal. “He got the priorities messed up. It’s supposed to be patients first.”

Missing Paperwork

Meanwhile, employees who requested the paperwork to file for workers’ comp were repeatedly ignored, according to Reveal.

“I just knew after the third or fourth time that they weren’t going to do anything about it,” a former employee whose back was crushed under a falling Model X hatchback told Reveal. “I was very frustrated. I was upset.”

The automaker is on the hook for up to $750,000 in medical payments per workers’ comp claim, according to Reveal‘s reporting.

Meanwhile, both Tesla CEO Elon Musk and Laurie Shelby, the company’s VP of safety, have publicly praised Access Omnicare, Reveal found. Musk even recently announced plans to extend it to other plants, “so that we have really immediate first-class health care available right on the spot when people need it.”

READ MORE: How Tesla and its doctor made sure injured employees didn’t get workers’ comp [Reveal News]

More on Tesla: Video Shows Tesla Autopilot Steering Toward Highway Barriers

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Report: Tesla Doc Is Playing Down Injuries to Block Workers’ Comp

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 were conducted, with 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 initiated in 1993. The goal was to cure malignant brain tumors by using recombinant DNA to transfer a gene making the tumor cells sensitive to a drug that in turn would cause the tumor cells to die.[10]

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.[11] These include treatment of retinal diseases Leber’s congenital amaurosis[12][13][14][15] and choroideremia,[16] X-linked SCID,[17] ADA-SCID,[18][19] adrenoleukodystrophy,[20] chronic lymphocytic leukemia (CLL),[21] acute lymphocytic leukemia (ALL),[22] multiple myeloma,[23] haemophilia,[19] and Parkinson’s disease.[24] Between 2013 and April 2014, US companies invested over $600 million in the field.[25]

The first commercial gene therapy, Gendicine, was approved in China in 2003 for the treatment of certain cancers.[26] 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.[27]In 2012 Glybera, a treatment for a rare inherited disorder, Lipoprotein lipase deficiency became the first treatment to be approved for clinical use in either Europe or the United States after its endorsement by the European Commission.[11][28]

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.[29] 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.[28]

DNA must be administered, reach the damaged cells, enter the cell and either express or disrupt a protein.[30] Multiple delivery techniques have been explored. The initial approach incorporated DNA into an engineered virus to deliver the DNA into a chromosome.[31][32] Naked DNA approaches have also been explored, especially in the context of vaccine development.[33]

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.[34]

Gene editing is a potential approach to alter the human genome to treat genetic diseases,[35] viral diseases,[36] and cancer.[37] As of 2016[update] these approaches were still years from being medicine.[38][39]

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.[40] [needs update]

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[41] prohibit GGT for application in human beings, for technical and ethical reasons, including insufficient knowledge about possible risks to future generations[41] and higher risks versus SCGT.[42] The US has no federal controls specifically addressing human genetic modification (beyond FDA regulations for therapies in general).[41][43][44][45]

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.[52] 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.[53]

In 1972 Friedmann and Roblin authored a paper in Science titled “Gene therapy for human genetic disease?”[54] Rogers (1970) was cited for proposing that exogenous good DNA be used to replace the defective DNA in those who suffer from genetic defects.[55]

In 1984 a retrovirus vector system was designed that could efficiently insert foreign genes into mammalian chromosomes.[56]

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.[57] 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.[58]

Cancer gene therapy was introduced in 1992/93 (Trojan et al. 1993).[59] 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 24 November 1993,[60] 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.[61] 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.[62]

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.[63]

Jesse Gelsinger’s death in 1999 impeded gene therapy research in the US.[64][65] As a result, the FDA suspended several clinical trials pending the reevaluation of ethical and procedural practices.[66]

The modified cancer gene therapy strategy of antisense IGF-I RNA (NIH n 1602)[60] 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.[67] 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.[68]

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.[69]

Researchers created liposomes 25 nanometers across that can carry therapeutic DNA through pores in the nuclear membrane.[70]

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.[71]

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.[72]

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.[26]

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.[73]

In May a team reported a way to prevent the immune system from rejecting a newly delivered gene.[74] 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.[75]

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.[76][77]

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.[78]

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.[12] 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.[12][13][14][15]

In September researchers were able to give trichromatic vision to squirrel monkeys.[79] 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.[80]

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.[81]

In September it was announced that an 18-year-old male patient in France with beta-thalassemia major had been successfully treated.[82] Beta-thalassemia major is an inherited blood disease in which beta haemoglobin is missing and patients are dependent on regular lifelong blood transfusions.[83] 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.[84] 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.[84][85] Further clinical trials were planned.[86] Bone marrow transplants are the only cure for thalassemia, but 75% of patients do not find a matching donor.[85]

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).[87][88]

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.[89] 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.[21] In 2013, the researchers announced that 26 of 59 patients had achieved complete remission and the original patient had remained tumor-free.[90]

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.[91][92]

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.[93][27] Neovasculogen is a plasmid encoding the CMV promoter and the 165 amino acid form of VEGF.[94][95]

The FDA approved Phase 1 clinical trials on thalassemia major patients in the US for 10 participants in July.[96] The study was expected to continue until 2015.[86]

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.[97] The recommendation was endorsed by the European Commission in November 2012[11][28][98][99] and commercial rollout began in late 2014.[100] Alipogene tiparvovec was expected to cost around $1.6 million per treatment in 2012,[101] revised to $1 million in 2015,[102] making it the most expensive medicine in the world at the time.[103] As of 2016[update], only the patients treated in clinical trials and a patient who paid the full price for treatment have received the drug.[104]

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.[23]

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.[22]

Following encouraging Phase 1 trials, in April, researchers announced they were starting Phase 2 clinical trials (called CUPID2 and SERCA-LVAD) on 250 patients[105] 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.[106] The FDA granted this a Breakthrough Therapy Designation to accelerate the trial and approval process.[107] In 2016 it was reported that no improvement was found from the CUPID 2 trial.[108]

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.[109] The other children had Wiskott-Aldrich syndrome, which leaves them to open to infection, autoimmune diseases, and cancer.[110] Follow up trials with gene therapy on another six children with Wiskott-Aldrich syndrome were also reported as promising.[111][112]

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.[19] In 2014 a further 18 children with ADA-SCID were cured by gene therapy.[113] ADA-SCID children have no functioning immune system and are sometimes known as “bubble children.”[19]

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.[19][114]

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.[115][116] By 2016, 32 patients had been treated with positive results and researchers were hopeful the treatment would be long-lasting.[16] 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.[117][118]

Clinical trials of gene therapy for sickle cell disease were started in 2014.[119][120] 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.[121][needs update]

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.[122]

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.[123][124]

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”.[125][126][127][128]

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]).[129] Children with highly aggressive ALL normally have a very poor prognosis and Layla’s disease had been regarded as terminal before the treatment.[130]

In December, scientists of major world academies called for a moratorium on inheritable human genome edits, including those related to CRISPR-Cas9 technologies[131] but that basic research including embryo gene editing should continue.[132]

In April the Committee for Medicinal Products for Human Use of the European Medicines Agency endorsed a gene therapy treatment called Strimvelis[133][134] and the European Commission approved it in June.[135] 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.[136]

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.[137][138]

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.[139]

In February Kite Pharma announced results from a clinical trial of CAR-T cells in around a hundred people with advanced Non-Hodgkin lymphoma.[140]

In March, French scientists reported on clinical research of gene therapy to treat sickle-cell disease.[141]

In August, the FDA approved tisagenlecleucel for acute lymphoblastic leukemia.[142] 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.[143]

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.[144][145]

In December, the FDA approved Luxturna, the first in vivo gene therapy, for the treatment of blindness due to Leber’s congenital amaurosis.[146] The price of this treatment was 850,000 US dollars for both eyes.[147][148]

In February 2019, medical scientists working with Sangamo Therapeutics, headquartered in Richmond, California, announced the first ever “in body” human gene editing therapy to permanently alter DNA – in a patient with Hunter Syndrome.[149] Clinical trials by Sangamo involving gene editing using Zinc Finger Nuclease (ZFN) are ongoing.[150]

Speculated uses for gene therapy include:

Athletes might adopt gene therapy technologies to improve their performance.[151] 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.[152]

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.[153][154][155] For parents, genetic engineering could be seen as another child enhancement technique to add to diet, exercise, education, training, cosmetics, and plastic surgery.[156][157] Another theorist claims that moral concerns limit but do not prohibit germline engineering.[158]

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.”[159]

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,[160] and such concerns have continued as technology progressed.[161][162] 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.[125][126][127][128] In April 2015, researchers sparked controversy when they reported results of basic research to edit the DNA of non-viable human embryos using CRISPR.[163][164] A committee of the American National Academy of Sciences and National Academy of Medicine gave qualified support to human genome editing in 2017[165][166] once answers have been found to safety and efficiency problems “but only for serious conditions under stringent oversight.”[167]

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.[168]

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.[169]

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.[170] 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.[171] 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.[170]

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.[172][173]

Gene therapy is the basis for the plotline of the film I Am Legend[174] and the TV show Will Gene Therapy Change the Human Race?.[175] In 1994, gene therapy was a plot element in “The Erlenmeyer Flask”, the first season finale of The X-Files; it is also used in Stargate as a means of allowing humans to use Ancient technology.[176][177]

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Gene therapy – Wikipedia

Gene Therapy – REGENXBIO

A change or damage to a gene can affect the message the gene carries, and that message could be telling our cells to make a specific protein that the body needs in order to function properly. NAV Gene Therapy focuses on correcting these defects in genetic diseases by delivering a healthy, working copy of the gene to the cells in need of repair, which potentially enables the body to make the deficient protein. The NAV Technology Platform can also be used to deliver a gene that allows the body to produce a therapeutic protein to treat a specific disease.

Heres how the NAV Technology Platform works:

First, our scientists insert the gene of interest (that is, either the missing/defective gene or a gene to create a therapeutic protein) into a NAV Vector. A NAV Vector is a modified adeno-associated virus (AAV), which is not known to cause disease in humans. It is common for viruses to be used as vectors in gene and cell therapy. The NAV Vector acts as a delivery vehicle, transporting and unloading the gene into cells where the gene triggers production of the protein the body needs.

Our NAV Technology Platform includes more than 100 novel AAV vectors, including AAV8, AAV9 and AAVrh10, many of which are tailored to reach specific areas of the body where the gene is needed most. For example, gene therapy delivered to the liver has the potential to treat metabolic diseases like hemophilia, whereas gene therapy designed to reach the central nervous system (brain and spinal cord) may primarily impact symptoms of diseases that affect the brain and cognition.

Next, the NAV Vector is administered into the patient by injection or infusion, and is expected to make its way to cells that need the protein. The NAV Vector is designed to reach the target cells and deliver the gene it is carrying, enabling the cells to make the protein the body needs. These genes have the potential to correct disease by triggering production of a therapeutic protein or by allowing the bodys natural mechanisms to work the way they were intended.

Because gene therapies may have a long-term effect, a single administration of NAV Gene Therapy has the potential to do the same work as years of conventional chronic therapies.

Learn more about gene therapy below:

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Gene Therapy – REGENXBIO

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

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

Gene Therapy – Sickle Cell Anemia News

Gene therapy is an experimental technique that aims to treat genetic diseases by altering a disease-causing gene or introducing a healthy copy of a mutated gene to the body. The U.S. Food and Drug Administrationapprovedthe first gene therapy for an inherited disease a genetic form of blindness in December 2017.

Sickle cell anemia is caused by a mutation in the HBB gene which provides the instructions to make part of hemoglobin, the protein in red blood cells that carries oxygen.

Researchers are working on two different strategies to treat sickle cell anemia with gene therapy. Both of these strategies involve genetically altering the patients own hematopoietic stem cells. These are cells in the bone marrow that divide and specialize to produce different types of blood cells, including the red blood cells.

One strategy is to remove some of the patients hematopoietic stem cells, replace the mutated HBB gene in these cells with a healthy copy of the gene, and then transplant those cells back into the patient. The healthy copy of the gene is delivered to the cells using a modified, harmless virus. These genetically corrected cells will then hopefully repopulate the bone marrow and produce healthy, rather than sickled, red blood cells.

The other strategy is to genetically alter another gene in the patients hematopoietic stem cells so they boost production of fetal hemoglobin a form of hemoglobin produced by babies from about seven months before birth to about six months after birth. This type of hemoglobin represses sickling of cells in patients with sickle cell anemia, but most people only produce a tiny amount of it after infancy. Researchers aim to increase production of fetal hemoglobin in stem cells by using a highly specific enzyme to cut the cells DNA in the section containing one of the genes that suppress production of fetal hemoglobin. When the cell repairs its DNA, the gene no longer works and more fetal hemoglobin is produced.

Gene therapy offers an advantage over bone marrow transplant, in that complications associated with a bone marrow donation now the only cure for the disease such as finding the right match are not a concern.

Twelve clinical trials studying gene therapy to treat sickle cell anemia are now ongoing. Nine of the 12 are currently recruiting participants.

Four trials (NCT02186418, NCT03282656, NCT02247843, NCT02140554) are testing the efficacy and safety of gene therapy to replace the mutated HBB gene with a healthy HBB gene. These Phase 2 trials are recruiting both children and adults in the United States and Jamaica.

Three trials (NCT02193191, NCT02989701, NCT03226691) are investigating the use ofMozobil (plerixafor) in patients with sickle cell anemia to increase the production of stem cells to be used for gene therapy. This medication is already approved to treat certain types of cancer. All three are recruiting U.S. participants.

One trial (NCT00669305) is recruiting sickle cell anemia patients in Tennessee to donate bone marrow to be used in laboratory research to develop gene therapy techniques.

The final study(NCT00012545) is examining the best way to collect, process and store umbilical cord blood from babies with and without sickle cell anemia. Cord blood contains abundant stem cells that could be used in developing gene therapy for sickle cell anemia. This trial is open to pregnant women in Maryland both those who risk having an infant with sickle cell anemia, and those who do not.

One clinical trial (NCT02151526) conducted in France is still active but no longer recruiting participants. It is investigating the efficacy of gene therapy in seven patients. For the trial, a gene producing a therapeutic hemoglobin that functions similarly to fetal hemoglobin is introduced into the patients stem cells. A case studyfrom one of the seven was published in March 2017; it showed that the approach was safe and could be an effective treatment option for sickle cell anemia.

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Gene Therapy Net – News, Conferences, Vectors, Literature …

Posted on: 28 November 2018, source: fortune.comResearchers in China (Clinical project ‘Safety and validity evaluation of HIV immune gene CCR5 gene editing in human embryos’) used genetic engineering tools (CRISPR) to create twins theoretically immune to HIV, smallpox, and cholera, MIT Technology Review reported. The medical breakthrough is controversial, as many worry about eugenics and designer babies for the wealthy. The twins, named Lulu and Nana, according to lead scientist He Jiankui of Shenzhen in a YouTube video, were the result of in vitro fertilization (IVF). A few weeks old, they appear to be healthy. When they were a single cell, genetic surgery using a popular tool, CRISPR, removed the doorway through which HIV enters to infect people.

Watch the presentation of He Jiankui and discussion during Second International Summit on Human Genome Editing in Hongkong, Wednesday November 28th, 2018.

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Gene Therapy Net – News, Conferences, Vectors, Literature …