Gene therapy for sickle cell disease passes key preclinical test

A precision-engineered gene therapy virus, inserted into blood stem cells that are then transplanted, markedly reduced sickle-induced red-cell damage in mice with sickle cell disease, researchers from Dana-Farber/Boston Children’s Cancer and Blood Disorders Center reported in the Journal of Clinical Investigation.

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Sickle cell gene therapy passes key preclinical test - Boston Children's Answers

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BCL11A-based gene therapy for sickle cell disease passes key preclinical test

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