Defining the therapeutic window in a severe animal model of spinal muscular atrophy

Spinal muscular atrophy (SMA) is a neurodegenerative disease caused by the loss of a single gene, Survival Motor Neuron-1 (SMN1). Administration of a self-complementary Adeno-Associated Virus vector expressing full-length SMN cDNA (scAAV-SMN) has proven an effective means to rescue the SMA phenotype...

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Main Authors: Robbins, Kate L., Glascock, Jacqueline J., Osman, Erkan Y., Miller, Madeline R., Lorson, Christian L.
Formato: Artigo
Idioma:Inglês
Publicado: Oxford University Press 2014
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Articles
Acceso en liña:https://ncbi.nlm.nih.gov/pmc/articles/PMC4119406/
https://ncbi.nlm.nih.gov/pubmed/24722206
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1093/hmg/ddu169
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