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Dr. Gary Gibbons and Dr. Julie Panepinto
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FDA approval of gene therapies for sickle cell disease: Q&A with NHLBI Director Dr. Gary Gibbons and NHLBI’s Division of Blood Diseases and Resources Director Dr. Julie Panepinto
The U.S. Food and Drug Administration approved two new gene therapies to treat sickle cell disease — one a gene-editing and the other a gene-addition approach. Nearly 100,000 people in the United States — and millions worldwide — have been diagnosed with this painful, life-threatening genetic blood disease. To help explain the historic importance...
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