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FDA approves 2 gene therapies for sickle cell. One is the first to use the editing tool CRISPR

Por: WPLG Local 10 Health December 09, 2023

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Regulators on Friday approved two gene therapies for sickle cell disease that doctors hope can cure the painful, inherited blood disorder that afflicts mostly Black people in the U.S.The Food and Drug Administration said the one-time treatments can be used for patients 12 and older with severe forms of the disease. One, made by Vertex Pharmaceuticals and CRISPR Therapeutics, is the based on CRISPR, the gene editing tool that won the Nobel Prize... + full article



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