Scientists successfully edited a human embryo to erase a heart condition

First Human Embryos Edited in US

Scientists from Oregon Health and & Science University used the gene-editing tool CRISPR to target the mutation that causes hypertrophic cardiomyopathy, a common genetic heart disease that can cause sudden cardiac death and heart failure. The consequences of the research may not be felt for quite a while yet as clinical trials remain a long way off, and are in fact now impermissible under federal law. Though it's still in the testing phase, CRISPR has been likened to eugenics or playing God by allowing scientists to create "designer babies" or wipe out diseases through the kind of genetic engineering now limited to plants and lower animals.

This progress has come over the attempts by the federal government to prevent it by not funding any research that leads to changes in the "germ line" - sperm, cells etc., that transmit genetic information.

An account of the research, complete with photos of the multiplying cells, was published in the prestigious British journal "Nature".

"Really, we didn't edit anything, neither did we modify anything", explained Shoukhrat Mitalipov, lead author and a researcher at the Oregon Health and Science University.

"What our report said was, once the technical hurdles are cleared, then there will be societal issues that have to be considered and discussions that are going to have to happen".

For the first time, researchers in the USA have safely repaired a disease-causing gene in human embryos, targeting a heart defect best known for killing young athletes - a big step toward one day preventing a list of inherited diseases. Previously, off-target edits with CRISPR have made human embryos unviable for clinical use.

Along with the man's sperm, researchers injected into the egg the DNA-cutting enzyme Cas9 and a piece of RNA to direct the enzyme to snip the mutant copy of the gene.

Basic or not, the development is remarkable."By using this technique, it's possible to reduce the burden of this heritable disease on the family and eventually the human population", Mitalipov said in an OHSU press release.

But this prospect is still distant: "additional research, as well as an ethical debate will be needed before clinical trials", has taken care to specify the professor Amato.

"Overall, this is a very cool and surprising result, especially that it worked as well as it did", stem cell scientist Paul Knoepfler of the University of California, Davis, told BuzzFeed News.

Potentially, it could apply to any of more than 10,000 conditions caused by specific inherited mutations.

Moreover, there are thousands of genetic diseases, ranging from cystic fibrosis to sickle cell anemia, for which such a procedure could be relevant.

"You could quite imagine that in the future the demand would increase".

"It is a very important study", said Dr. Dusanka Babovic-Vuksanovic, genomics chair at Mayo Clinic in Rochester, Minnesota. Carrying just one mutant copy of the gene causes an inherited heart problem called hypertrophic cardiomyopathy (SN: 9/17/16, p. 8).

He said: "Every generation on would carry this fix because we've removed the disease-causing gene variant from that family's lineage".

The research significantly improves upon previous efforts.

In February a National Academy of Sciences, Engineering and Medicine committee endorsed modifying embryos, but only to correct mutations that cause "a serious disease or condition" and when no "reasonable alternatives" exist.

For now, the Germany-based researcher Adjaye is skeptical that of the idea that the technique could be used to create so-called designer babies.

But these embryos weren't actually allowed to develop beyond a few days.

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