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Showing articles 0 to 3 of 3 Filter Applied: gene therapy (Click to remove) Muscular Dystrophies Lancet 394:2025-2038, Mercuri, E.,et al, 2019 Hematopoietic Stem - and Progenitor-Cell Gene Therapy for Hurler Syndrome NEJM 385:1929-1940, Gentner, B.,et al, 2021 Single-Dose Gene-Replacement Therapy for Spinal Muscular Atrophy NEJM 377:1713-1722,1786, Mendell, J.R.,et al, 2017 Showing articles 0 to 3 of 3