CRISPR to be used to edit human genes in the US?

June 21, 2016  10:42

A federal panel will get its first look next week at a proposal to use the revolutionary gene-editing technology CRISPR in humans, in this case to try to treat cancer.

The experiment, proposed by the University of Pennsylvania, would remove certain cells of the immune system, called T cells, from cancer patients. Scientists would then use CRISPR to genetically modify the T cells so that when they’re returned to the patient, they target and destroy myeloma, melanoma, and sarcoma tumor cells.

Carrie Wolinetz, the associate director for science policy at the National Institutes of Health, disclosed the review in a blog post, which was first reportedby MIT Technology Review. The federal panel must review all human experiments that alter DNA.

A biotech company, Editas Medicine in Cambridge, Mass., had been expected to be the first to use CRISPR in people, in an effort to treat a rare eye disease. But it looks like Penn might beat Editas to the punch.

Neither Penn nor Editas immediately responded to requests for comment.

Penn scientist Dr. Carl June pioneered the use of genetically modified T cells, called CAR-T’s, as a cancer treatment. So far, researchers have used traditional genetic engineering to make the cells home in on the molecules that stick out of tumor cells. But that’s less efficient and often less precise than CRISPR.

Penn is reportedly seeking NIH approval to use CRISPR to edit out two genes in T cells. One, called PD-1, suppresses the ability of T cells to attack tumors. The other is a T cell receptor that can turn the body’s natural defenses against itself.

The medical significance of the proposal is not clear, since gene editing has already been used in humans — just not gene editing via the CRISPR-Cas9 system.

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