Published: Fri, August 04, 2017
Medical | By Dorothy Lyons

Scientists Successfully Correct Faulty Gene in Human Embryos

Scientists Successfully Correct Faulty Gene in Human Embryos

Scientists at the Salk Institute in La Jolla have helped to edit the DNA of human embryos for the first time in the United States.

Last week, genetic biologist Shoukhrat Mitalipov and a team of researchers at Oregon Health and Science University successfully edited the genes of 167 human embryos using the medical tool CRISPR-Cas9.

An global group of 11 organizations Wednesday issued a political statement against the changes to the genome ends with implantation and pregnancy, at the same time supporting research, financed from the state budget, the potential clinical applications.

The researchers said more work is needed to flawless the technique and see how widely it could be applied. "We need to be sure this can be done reproducibly and effectively". The mutation associated with hypertrophic cardiomyopathy is in the MYBPC3 gene.

The scientists focused on hypertrophic cardiomyopathy, the most common cause of sudden death in otherwise healthy young athletes.

It's far too early to use the technique as a treatment for the disease, which affects about 1 in 500 people. The embryos were created from eggs donated by healthy women, which were artificially inseminated with sperm from males carrying the mutation.

Mitalipov aims to continue his research, published Wednesday in the journal Nature. The Salk scientists contributed by developing the gene editing strategy, initially testing it in stem cells derived from the patient's skin cells. But they found that their edits were imprecise.

A similar experiment had been conducted in China in 2015, but with mixed results.

Professor Peter Braude, from King's College London, said: "With this paper the possibility of germline genome editing has moved from future fantasy to the world of possibility, and the debate about its use, outside of fears about the safety of the technology, needs to run to catch up".

The scientists were surprised by just how safe and efficient the method was.

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"Looking at it more closely, it's less useful than you might expect, if it works at all", Greely said.

This image shows individual blastomeres within the early embryos two days after co-injection.

Crispr makes it possible to manipulate a genome in the way that we can alter the words in a word processor.

And some embryos had cells that did not get repaired-a phenomenon called mosaicism that could result in the mutation being passed on-as well as unplanned mutations that could cause other health problems. In addition, there are thousands of genetic diseases wherein the same procedure could be applied. Or scientists can try delivering the missing DNA in a fix package, like a computer's cut-and-paste program.

However, permitting edits to germ line cells - such as embryos, eggs, and sperm - could also be "very risky on multiple levels", DiCamillo warned. Their work was largely funded by private donations and university money. Under normal conditions, half of the man's children would inherit the gene, the disease and the risk for passing it on. They had a 72 percent success rate in the gene correction.

"One thing clear at this stage is that we shouldn't apply this technology in any shape or form toward designer babies", Wu said. And even if it was there, there is still a lot of ethical considerations and scientific limitations.

What lies ahead for gene-editing research? The new study abides by those recommendations.

Mitalipov added that he believes it's "unlikely" that the technique would be used for genetic modification.

It's important to note that for now, this research is only being done in the lab - scientists aren't ready to attempt this kind of genetic adjustment in the womb yet.

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