Monthly Archives: August 2017

CRISPR-Cas9 gene editing has captured the publics imagination. As this powerful technology becomes even more popular, it has also incited plenty of fears about what the future may bring. But a closer look at recent milestones and studies demonstrate the future of gene editing is already happening right now.

This experimental technique, known as CRISPR (pronounced crisper) for short,utilizes snippets of bacteria as a pair of molecular scissors. The technology allows scientists to selectively modify DNA segments, disable or alter genes or correct mutations in the genome of any living organism. In a controversial landmark study published earlier this month in Nature, scientists eliminated a genetic abnormality in a human embryo.

Gene editing is proving to be a nimble and versatile technology for redesigning the world. This area of research is certain to change nearly every field of biological sciencesincluding agriculture, medicine and zoologyand touch every aspect of our lives. Here are 10 ways scientists have already used CRISPR gene editing to do what once seemed impossible.

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Here's how CRISPR-Cas9 works. REUTERS

A potential cure for diabetes. Scientists created genetically modified skin grafts to protect lab mice from diabetes. The experiment could help researchers identify a suitable substitute for insulin.

Eliminated disease from mosquitoes. In one experiment, researchers bred mosquitos that are resistant to the parasite that causes malaria.

Created a new type of seafood. The U.S. Food and Drug Administration approved a genetically modified salmon, known as AquaAdvantage salmon. Gene editing gave the fish the taste and texture of Chinook salmon and the efficient, rapid growth of ocean pout. Canadians are already eating them.

Super-strength animals. By deleting the relevant gene, scientists in China bred goats with more muscle (for meat) and hair (for wool).

Several changes to pigs. Scientists edited out all traces of porcine endogenous retrovirus (PERV), which brings us one step closer to creating a sustainable organ supply for transplant patients. Another new strain of pigs, known as Enviropigs produces manure that is low in phosphorus.Scientists can also now cut off the gene responsible for growth hormone in the animals, which could make micropigs the next pet.

Treated cancer. CRISPR has been used in a number pilot studies in China to treat aggressive cancers. In astudyof head and neck cancer, scientists tweaked genetic mutations in a patients blood, andthen injected the blood back into thepatient in order to suppress tumor growth.

Bolster the wine supply. At Rutgers University, researchers developed a way to cultivate grapes that can resist a type of mildew that can spoil the crop.

Make antisocial ants. Ants rely on their keen sense of smell to communicate. When scientists edited out the gene responsible for their sense of smell, the bugs behaviors changed. The researchers found their antennae and brain circuits didnt fully develop. Productivity in the colony, such as food foraging, also went down dramatically because the bugs were unable to work together effectively.

Eliminate cattle dehorning. The practice of removing the horns from cattle is especially painful to the animal. It'salso costly and time-consuming for farmers. Some scientists used CRISPR to breed cows that dont have horns.

Disable HIV. Though not yet studied in humans, scientists used gene editing to excise the HIV virus from the genomes of mice.

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Ten Weird Ways Scientists Are Changing the World With Gene Editing - Newsweek

Each year, some 30,000 patients undergo transplant surgery to receive an organ from a donor. Transplant medicine saves lives, but not enough people are willing to donate. Patients cant rely on the generosity of fellow humans to replace a heart, kidney or lungs. According to the United Network for Organ Sharing (UNOS), one patient is added to the U.S. transplant waiting list every 10 minutes, and 20 people on the national list die each day.

For decades, scientists have been hoping to address the organ shortage in more innovative ways, namely by tweaking the innards of other mammals to make them compatible with humans. Successfulanimal-to-human transplants (also known as xenotransplantation) would create a sustainable organ supply.

Pigs are the strongest contender for xenotransplantation because they have organs similar in size and physiological function to those found in humans. But pig organs on their own arent suitable for transplant. Human immune systems would most definitely reject pig organs. But an even greater challenge is the risk of animal viruses infectinghumans. Pigs carry active porcine endogenous retrovirus, and it remains unclear whether these viruses could becommunicable or fatal in humans. PERV infection would be dangerous becausetransplant recipients are routinely put on immunosuppressant drugs that make it difficult to fight off any bacteria or viruses.

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If animal-to-human transplants can be achieved successfully, it would create a sustainable organ supply. Thanks to gene editing, this may be possible in the future. REUTERS

Nowa team of researchers affiliated with Harvard Medical School appear to have solved one of these problems. Not only have these scientistsmade a controversial possibilityanimal organs in humansmore likely, but theyve done so using a controversial technology: CRISPR-Cas9 gene editing.

Through gene editing, the team eliminated all traces of the PERV virus from the cell line and conducted in vitro fertilization. There are 25 strains of PERV, which is the only known active retrovirus found in pigs. In the study, published Thursday in the journal Science, biologist Luhan Yang and her team implanted the PERV-free embryos into surrogates. The fetuses did not become reinfected with the virus, and the newbornpiglets are the first animals born without PERV. Yangwho founded eGenesis a few years ago to harness advances in CRISPR-Cas9 for the worldwide organ shortagewill now monitor the animals for any long-term effects.

Im a strong believer that science can help us improve health care if we look holistically for a solution, says Yang, lead author on the paper and chief science officer of eGenesis, the biotechnology company funding advancements in the research. Because there are millions of patients who suffer from end-stage organ failure, their life could potentially be saved, or largely improved, by this potential organ resource.

CRISPR-Cas9, or CRISPR (pronounced crisper) for short, is an experimental biomedical technique. The technology utilizes snippets of certain bacteria that allow for selective modifications of DNA segments, such as changingthe misspellings of a gene that contributeto mutations. Since CRISPR was identified several years ago, scientists have been using it in the laboratory to alter the genetic codes of living organisms. The new technologyis already leading advances once considered the stuff of science fiction. In astudy published last week in Nature,scientistseliminated a genetic abnormality in a human embryo.

Yang has been determined tousegene editing to solve the organ shortage problemfor several years. In 2013, sheand her team published the first paper showing CRISPR could be used to accurately and effectively alter the immune system. In 2015, she eradicated 62 copies of the PERV virus from a pig cancer cell line, which she says is a world record for researchers using CRISPR. The next step, she says, is to tweak the porcine genome further to prove the organs can be compatible with the human immune system.

Resurrecting aScientific Vision

For decades, xenotransplantation research seemed impossibly dangerous and financially risky both for small biomedical companies and large pharmaceutical companies. In the early 2000s, Novartis stopped funding xenotransplantation research. The U.S. Food Administration, fearing a public health disaster, began placing regulations on research facilities, whichmade studies even more challenging. The projects were costly andtoo complicated, and animal rights activists frequentlytargeted the scientists. But CRISPR is reviving the area of research once again, says Yang.

Transgenic PERV-free pigs could provide a source for solid organs as well as islet cells, which are tiny cells scattered throughout the pancreas that secrete insulin. Some successful pilot studies looked at porcine islet cell transfusions as a potential treatment for diabetes.

Dixon Kaufman, president-elect of the American Society of Transplant Surgeons and a transplant surgeon at the University of Wisconsin School of Medicine and Public Health, says its only a matter of timeprobably a few yearsbefore xenotransplant studies are open to patients. I think it is a realistic, almost palpable opportunity, he says. Anything that will improve safety, such as deleting this risk of the PERV infection, makes this more viable.

Kaufman thinks kidneys and pancreases will be the first solid animal organs transplanted into humans. Because these are non-vital organs, failure wouldnt necessarily lead to death. Patients who need a kidney could still receive dialysis, and those who need a pancreas could still access insulin.

These advances are a boon for transplant surgeons like Kaufman, who regularly have to tell patientsthey will probably die before a donated organ becomes available. He doesnt think a pig organ would be a hard sell to most of these patients, who are otherwise facing certain death.

The field is inherently sort of risky to begin with, and I think a lot of patients have already processed that, he says. I tell patientsin the grand designwe were not meant to swap body parts between ourselves.

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How Gene Editing And Pig Organs Can End The Human Transplant Shortage - Newsweek