Gene therapy for sickle cell disease passes key preclinical test
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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.
Stuart Orkin, Researchers
Archives — Page 198 of 873 — Harvard Gazette
Health Archives — Page 34 of 107 — Harvard Gazette
Archives — Page 198 of 873 — Harvard Gazette
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Archives — Page 198 of 873 — Harvard Gazette
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BCL11A-based gene therapy for sickle cell disease passes key preclinical test
