#gene therapy — Thursday, March 26, 2026 (2 items)
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AAV9 Gene Therapy in Type II GM1 Gangliosidosis - A Phase 1-2 Trial.
(nejm.org)
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New England Journal of Medicine 2026-03-26 | #clinical trial #neurodegeneration #gene therapy #lysosomal storage disease #aav vectors | |
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Prime Editing for p47(phox)-Deficient Chronic Granulomatous Disease.
(nejm.org)
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New England Journal of Medicine 2026-03-26 | #gene therapy #hematopoietic stem cells #immunodeficiency #base-editing |