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U.S. approves first gene therapy for inherited disease
JAY GORY, MANAGING EDITOR, science
The U.S. Food and Drug Administration on Tuesday approved Spark Therapeutics Inc's treatment for a rare form of blindness, marking the first time the agency has approved a gene therapy for an inherited disease. The drug, Luxturna, treats inherited retinal disease caused by defects in a gene known as RPE65, which tells cells to produce an enzyme critical for normal vision. Luxturna works by delivering 150 billion viral vector particles containing a correct copy of the RPE65 gene to retinal cells, restoring their ability to make the needed enzyme. read more


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