Research ArticleSpinal Muscular Atrophy

Systemic Delivery of scAAV9 Expressing SMN Prolongs Survival in a Model of Spinal Muscular Atrophy

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Science Translational Medicine  09 Jun 2010:
Vol. 2, Issue 35, pp. 35ra42
DOI: 10.1126/scitranslmed.3000830

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