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Gene editing spurs hope for transplanting pig organs into humans

11 August 2017

In a step toward making pigs suitable organ donors for humans, researchers at a startup company called eGenesis have for the first time used gene editing to eliminate a family of viruses in pigs that can be transmitted to people. "Even if organs from these gene-edited pigs could be safely used to overcome virus transmission, there remain formidable obstacles in overcoming immunological rejection and physiological incompatibility of pig organs in humans".

More than 117,000 Americans are now on a transplant wait-list and 22 people die every day awaiting a match, according to federal figures. In 2016, there were a record-high 33,500 transplants, but an estimated 22 people die each day waiting on a new organ. Getting organs from animals - particularly from pigs, whose organs tend to be close in size and work similarly to human organs - could be the solution to that shortage. But nearly all attempts to implant animal organs into people have failed. United Therapeutics is also going after xenotransplantation, while others are taking a different approach of trying to grow human organs in pigs. The virus, which is part of the pigs' DNA, has been an issue for human-pig transplants in the past because of concerns that it could infect humans. For example, the person's immune system can reject the animal organ, leading to death. The viruses the researchers targeted, which dwell in pig DNA and can be passed down during transplantation and infect human cells, have been another concern.

George Church, a Harvard Medical School geneticist who co-founded eGenesis and is a co-author of the paper, said piglets would need to be roughly four months old for their organs to be used for transplants.

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Luhan Yang of Boston biotech firm eGenesis and colleagues have previously shown they could use the highly efficient gene-editing technique CRISPR to disable the PERVs in pig cells grown in a dish. They then implanted the PERV-free embryos into surrogate sows and demonstrated the absence of PERV re-infection, initially in fetuses and finally in recently born piglets.

Piglets cloned from the fibroblast (connective tissue) cells turned out to be virus-free.

That hurdle may now have been cleared away, according to new research reported in the journal Science.

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If pig organs were shown to be safe and effective, "they could be a real game changer", said Klassen, who was not involved in the new study. These PERVs have the potential to infect humans if a pig organ is transplanted into a person, possibly causing tumors or leukemia. Since these kinds of viruses diseases have existed in animals for millions of years, scientists have thought removing them might have other unexpected negative consequences. Pigs are the biggest animals that have undergone CRISPR, he says, and he wants to see what happens when they are allowed to "grow to a ripe old age" of over 20.

A scientific advance using genetically edited piglets could lower the fatality rate and make using pig organs, similar to our own, a common practice.

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Gene editing spurs hope for transplanting pig organs into humans