Efficacy and biodistribution analysis of intracerebroventricular administration of an optimized scAAV9-SMN1 vector in a mouse model of spinal muscular atrophy
Spinal muscular atrophy (SMA) is an autosomal recessive disease of variable severity caused by mutations in the SMN1 gene. Deficiency of the ubiquitous SMN function results in spinal cord α-motor neuron degeneration and proximal muscle weakness. Gene replacement therapy with recombinant adeno-associ...
Main Authors: | , , , , , , , , , , , |
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Format: | Article |
Language: | English |
Published: |
Elsevier
2016-01-01
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Series: | Molecular Therapy: Methods & Clinical Development |
Online Access: | http://www.sciencedirect.com/science/article/pii/S2329050116301899 |