Sometimes, research delivers miracles
Posted on: October 11, 2017 at 12:03:29 CT
Silas MU
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On Thursday, U.S. Food and Drug Administration advisers will consider whether to recommend approval of a gene therapy that improved vision for these three youths and some others with hereditary blindness.
It would be the first gene therapy in the U.S. for an inherited disease, and the first in which a corrective gene is given directly to a patient. Only one gene therapy is sold in the U.S. now, a cancer treatment approved in August that engineers patients' blood cells in the lab.