Molecular Therapy
Volume 28, Issue 8, 5 August 2020, Pages 1887-1901
Original ArticleAAV9-Mediated Expression of SMN Restricted to Neurons Does Not Rescue the Spinal Muscular Atrophy Phenotype in Mice
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open access
Graphical Abstract
Keywords
SMA
AAV
gene therapy
SMN
neurons
peripheral organs
spinal muscular atrophy
synapsin promoter
ubiquitous promoter
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