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Scientists show that gene editing can 'turn off' human diseases

Engadget logo Engadget 11/30/2015 Jon Fingas
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Gene editing has already been used to fight diseases, but there's now hope that it might eliminate the diseases altgether. Researchers have shown that it's possible to eliminate facial muscular dystrophy using a newer editing technique, CRISPR (Clusters of Regularly Interspaced Short Palindromic Repeats) to replace the offending gene and 'turn off' the condition. The approach sends a mix of protein and RNA to bind to a gene and give it an overhaul.

This doesn't mean that doctors suddenly have a cure-all on their hands. They haven't tried CRISPR on real live people, and there's no guarantee that it'd work with every genetic condition under the Sun. The initial test was only 50 percent effective, too. If this gene mending is useful in the field, though, it could do a lot to transform medicine. Doctors could treat the root cause of a genetic disease rather than deal with the symptoms, and possibly wipe it out entirely -- or at least, make it more bearable.

[Image credit: Getty Images]

Molecular Therapy



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