CRISPR Cas9

Seven years after CRISPR-Cas9 gene editing appeared, clinical trials have begun.  The University of Pennsylvania has treated two patients, one with multiple myeloma, and the other with a sarcoma.  Other trials are in progress for sickle cell anemia, beta thalassemia, and an eye disease, Leber congenital amaurosis 10. When the first clinical gene therapy trial was approved in 1990 such experimental therapy fell under review of the Recombinant DNA Advisory Committee at NIH.  This year, to reflect broader oversight, the RAC was renamed the Novel and Experimental Technology and Research Advisory Committee.  We’ll see “what’s in a name.”  MORE

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