CNS-targeted gene therapy improves survival and motor function in a mouse model of spinal muscular atrophy

Spinal muscular atrophy (SMA) is a neuromuscular disease caused by a deficiency of survival motor neuron (SMN) due to mutations in the SMN1 gene. In this study, an adeno-associated virus (AAV) vector expressing human SMN (AAV8-hSMN) was injected at birth into the CNS of mice modeling SMA. Western bl...

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Autores principales: Passini, Marco A., Bu, Jie, Roskelley, Eric M., Richards, Amy M., Sardi, S. Pablo, O’Riordan, Catherine R., Klinger, Katherine W., Shihabuddin, Lamya S., Cheng, Seng H.
Formato: Artigo
Lenguaje:Inglês
Publicado: American Society for Clinical Investigation 2010
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Research Article
Acceso en línea:https://ncbi.nlm.nih.gov/pmc/articles/PMC2846065/
https://ncbi.nlm.nih.gov/pubmed/20234094
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1172/JCI41615
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