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Hematopoietic Stem - and Progenitor-Cell Gene Therapy for Hurler Syndrome
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Single-Dose Gene-Replacement Therapy for Spinal Muscular Atrophy
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Long-Term Effects of Antidarsagene Autotemcel for Metachromatic Leukodystrophy
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Muscular Dystrophies
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Spinal Muscular Atrophy A Timely Review
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