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...

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發表在:Mol Ther Methods Clin Dev
Main Authors: Armbruster, Nicole, Lattanzi, Annalisa, Jeavons, Matthieu, Van Wittenberghe, Laetitia, Gjata, Bernard, Marais, Thibaut, Martin, Samia, Vignaud, Alban, Voit, Thomas, Mavilio, Fulvio, Barkats, Martine, Buj-Bello, Ana
格式: Artigo
語言:Inglês
出版: Nature Publishing Group 2016
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Article
在線閱讀:https://ncbi.nlm.nih.gov/pmc/articles/PMC5022869/
https://ncbi.nlm.nih.gov/pubmed/27652289
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/mtm.2016.60
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