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Editas Medicine and Allergan are kicking off the first clinical trial of in vivo CRISPR gene editing, in which the editing of DNA takes place inside a person’s body. The trial will test CRISPR’s ability to remove a genetic mutation in people with a form of progressive childhood blindness. Genetic instructions for making the CRISPR-Cas9 machinery will be encoded into viruses, which will be injected under the retina. Another pair of companies—Crispr Therapeutics and Vertex Pharmaceuticals—have begun testing ex vivo CRISPR gene editing for inherited blood diseases.
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