Biotech and Pharma

Gene editing removed a big obstacle to transplanting organs from pigs to people

Key Points
  • Transplanting organs from pigs to humans could save lives
  • Process has some major risks
  • CRISPR editing appears to have solved one big problem
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A team of researchers say they have brought scientists one step closer to safely transplanting organs from pigs into humans.

Researchers from a biotechnology startup eGenesis and several universities in China, Denmark and the United States, have used CRISP-Cas9 gene editing techniques to remove diseases that could be transmitted from pigs to humans during tissue transplants.

The research could help scientists use animals to solve the shortage of available cells, tissues, and organs, which impacts hundreds of thousands of patients around the world.

The team published their results Thursday in the journal Science.

The concept of using animals to grow transplantable material for humans is called "xenotransplantation," and the team said in their study they consider it a promising solution to the problem.

"What we are trying to do is create a world where there is no shortage of organs," said eGenesis Co-Founder and Chief Scientific Officer Luhan Yang in an interview with CNBC. Yang was one of the authors of the current paper.

There are currently more than 100,000 people in the United States awaiting organ transplants, and only 20 to 30 percent of them will ever receive one, she said. eGenesis, based in Cambridge, Massachusetts, is entirely focused on furthering xenotransplantion as a viable option.

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But currently, one of the main risks of these techniques is that diseases known as "porcine endogenous retroviruses (PERVs)" could be spread to people.

In their study, the team designed a CRISi PR-Cas9 construct that would shut down the parts of the pig genomes responsible for PERVs. Once they successfully engineered cells with all of the sites shut down, they used a process known as somatic nuclear transfer to implant the modified DNA in a pig egg, creating an embryo free of PERVs.

They then implanted the embryo in surrogate sows, resulting in healthy pigs that showed no signs of the viruses.

Whether or not they could actually produce a viable pig without these viruses was something of an uncertainty, Yang said. 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. But the team found no such negative effect in the pigs.

The next step for the team is to solve the problem of immune compatibility between humans and pigs — there is a risk that the human body would reject tissue introduced from another species.

eGenesis closed a $38 million series A funding round, so Yang said team members are "keeping their heads down" and focusing on their next research challenge.

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