August 21, 2017

Pioneering scientists edit genes in embryos to stop inherited diseases

03 August 2017, 09:25 | Katie Chavez

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"We've always said in the past gene editing shouldn't be done, mostly because it couldn't be done safely", said Richard Hynes, a cancer researcher at the Massachusetts Institute of Technology who co-led the committee.

"I'm very surprised", study co-author Jin-Soo Kim of Seoul National University, in a briefing for reporters on the already widely discussed and speculated-about study, a collaboration between US, Chinese, and South Korean researchers. It is directed to a specific location in the DNA and performs a cut-and-paste function, not unlike word-processing software. He notes, however, that may simply be an artifact of the particular gene he targeted; there may be coincidentally no parts of the genome that have similar sequences as the gene that CRISPR cut.

When this technology is applied to "germ cells" - the sperm and eggs - or embryos, it changes the germline.

"None of the embryos we generated in this study were for reproductive purposes but if they were, the idea is that they wouldn't carry this mutation so the parents wouldn't have to worry about transferring this to their children, said Mitalipov".

The human embryos before and after their genomes were edited.

Until now, reports of studies using CRISPR on humans had been from outside the country and the results were not good.

The embryos weren't allowed to develop beyond eight cells, a standard for laboratory research.

According to the release, "There were no off-target mutations".

As a result, as the embryos divided and grew, many repaired themselves using the non-edited genes from the genetic materials of the female donors, and in total, 72 percent of the cells that formed appeared to be corrected. That's encouraging for one potential use of CRISPR in the future as a way to correct inherited genetic disease, says Mitalipov, since the embryo seems to have a built-in, reliable way of repairing the injury caused by splicing out an abnormal gene. Like all those affected, he carried both a normal and a defective copy of the MYBPC3 gene so his sperm population was a 50:50 mix of normal and defective.

The process was tested on 18 lab-created embryos using sperm from the male donor and eggs donated by 12 healthy young women, the study said. The research team cleverly side-stepped mosaicism by using CRISPR at the same time as fertilising the egg, before its cells had begun dividing.

But with no laws prohibiting it and clear proof that it can be done, germline manipulation in embryos can very well continue in the U.S., whether it's considered responsible or not. In 13 embryos, only a portion of cells had been edited. However, the team that conducted the recent study argues that CRISPR can be used to augment this process and increase the probability of pregnancy. Though using the research as treatment is illegal there as well as the United States, the regulatory barriers are much higher in America and look unlikely to be changed.

Source – MIT Technology Review
Source – MIT Technology Review

The technique already has been used in animals for generating mutant models. Although still at the proof-of-concept phase, this new experiment suggests that the near-future will feature a way not to just "pre-emptively cure" a person of the disease, but remove it from their offspring forever.

Mitalipov's team may have finally found the fix. These were sequenced and the researchers found that the editing had indeed worked. "Now we have shown it is not going to be a big issue, I hope they will reconsider".

"We're rewriting the genome in a way which is inheritable across generations".

Still, the technology was not 100 percent successful. And even if it was there, there is still a lot of ethical considerations and scientific limitations.

In our view, it is unlikely that genome editing would be used to treat the majority of inherited conditions anytime soon.

The new procedure tackled a genetic mutation in human embryos that causes hypertrophic cardiomyopathy, an inherited condition in which the heart muscle becomes abnormally thick.

Illustration of a human embryo.

In Australia, the National Health and Medical Research Council has a strict set of guidelines, meaning that all research performed on human embryos is monitored very closely, and many limitations exist.

That doesn't mean that we shouldn't have a public debate about the ethical implications - it is hugely important that we do.

The study was not, however, the world-altering breakthrough it was made out to be after news of the forthcoming paper was leaked to the MIT Technology Review last week, and covered by most major media outlets, including Gizmodo.

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