A CRISPR trick in blind mice points the way to possible treatments for inherited diseases
Using CRISPR, scientists have edited the bad copy of a gene while sparing its healthy version, an advance that might help people with a common form of blindness and other…
by Sharon Begley
Feb 02, 2018
3 minutes
It might seem that scientists have never met a chunk of DNA they couldn’t edit in mice or isolated cells using CRISPR — from mutations causing deafness to those for Duchenne muscular dystrophy. In fact, they are learning what every pencil- or Word-wielding editor knows: It’s much easier to improve something that’s in terrible shape than writing that’s near perfect.
In genome-editing, the challenge for CRISPR-wielding scientists is to edit only one of the two copies, or alleles, of every gene that people have, repairing the ever-so-slightly broken one and leaving
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