Systemic Administration of a Recombinant AAV1 Vector Encoding IGF-1 Improves Disease Manifestations in SMA Mice

Spinal muscular atrophy is a progressive motor neuron disease caused by a deficiency of survival motor neuron. In this study, we evaluated the efficacy of intravenous administration of a recombinant adeno-associated virus (AAV1) vector encoding human insulin-like growth factor-1 (IGF-1) in a severe...

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Detalhes bibliográficos
Publicado no:Mol Ther
Main Authors: Tsai, Li-Kai, Chen, Chien-Lin, Ting, Chen-Hung, Lin-Chao, Sue, Hwu, Wuh-Liang, Dodge, James C, Passini, Marco A, Cheng, Seng H
Formato: Artigo
Idioma:Inglês
Publicado em: Nature Publishing Group 2014
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Original Article
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC4435595/
https://ncbi.nlm.nih.gov/pubmed/24814151
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/mt.2014.84
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