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By tucking two crucial sentences inside a federal spending bill last year, the U.S. Congress effectively banned the human testing of gene-editing techniques that could produce genetically modified babies. But the provision, which is up for renewal this year, has also flustered proponents of a promising technique that could help mothers avoid passing certain devastating genetic disorders to their children.

 

The language in the bill is a clear reference to the use of techniques like CRISPR to modify the human germline (see “Engineering the Perfect Baby”). Most scientists agree that testing germline editing in humans isirresponsible at this point. But regulators have decided that the description also fits mitochondrial replacement therapy, which entails removing the nucleus from a human egg and transplanting it into one from a different person to prevent the transmission of debilitating or even deadly mitochondrial disorders to children.

 

Mitochondria are the components of the cell responsible for producing energy. They also have their own DNA, separate from the DNA in the nucleus. Babies always inherit their mother’s mitochondrial genome. Mutations in the mitochondrial genome, in the nuclear genome, or both can lead to a wide range of mitochondrial disorders, many with severe and even debilitating symptoms. Between 1,000 and 4,000 children are born with mitochondrial diseases every year, and there are no licensed therapies or cures for these diseases.

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MIT Technology Review

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