Febrero 22, 2018

Scientists gene-edit piglets, bringing transplants to humans closer

11 Agosto 2017, 04:55 | Verda Sainz

Harvard geneticist George Church Credit Harvard

Harvard geneticist George Church

Animal organs could be transplanted into people within two years, according to a scientist who has modified a pig's genome to remove harmful viruses.

The news comes as more than 6,000 patients waiting for an organ transplant in the United Kingdom, according to Government figures.

The most genetically modified animals in existence have been created to help end a shortage of organs for transplant, say USA researchers.

They can move around within the genome, and in xenotransplantation, there is the possibility that they could jump from the transplanted organ into human cells - and, in doing so, disrupt the cells they integrate into, similar to what was seen with some early gene therapy vectors. In it, the scientists show how they were able to generate 37 designer pigs without active porcine endogenous retroviruses (PERVs) that can be transmitted to humans and are potentially deadly.

Worldwide, human organs for transplant are in short supply.

For the study, the researchers identified 25 genomic sites where PERVs present in the genome of pig fibroblast cells, and using CRISPR they deactivated all.

Pigs, whose organs are roughly the same size and shape as ours, have always been eyed as a possible source of functional transplants for humans, especially as transplant waiting lists continue to lengthen. However, other researchers argue that it could be years before scientists even know if pig organ transplants are safe. An estimated 22 people die each day waiting for an organ transplant.

If successful this could pave the way for organ transplants such as livers, hearts and other organs from pigs into humans. However, pigs also have many viruses embedded in their DNA, passed down the generations in sperm and eggs. Yang has also invented eGenesis, a few years back to advance the CRISPR-Cas9 technology in order to solve the organ shortage. 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. They allowed each egg to develop into an embryo and implanted it in the uterus of a surrogate mother.

The National Health and Medical Research Council (NHMRC) code for the care and use of animals for scientific purposes states that all teaching and research activities "must balance whether the potential effects on the wellbeing of the animals involved is justified by the potential benefits". They grew the resulting embryos in lab dishes and then transferred healthy ones to sows, hoping for pregnancies. When these pig cells grew in the laboratories, next to human embryonic kidney cells, these viruses spread to the human cells.

Dr. Church's company eGenesis, would be selling the genetically altered pig organs.

In fact, a team of scientists from the University of Alabama at Birmingham recently engineered pig organs that are free of carbohydrates, which typically make the organs targets for rejection.

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