Systemic, postsymptomatic antisense oligonucleotide rescues motor unit maturation delay in a new mouse model for type II/III spinal muscular atrophy

Clinical presentation of spinal muscular atrophy (SMA) ranges from a neonatal-onset, very severe disease to an adult-onset, milder form. SMA is caused by the mutation of the Survival Motor Neuron 1 (SMN1) gene, and prognosis inversely correlates with the number of copies of the SMN2 gene, a human-sp...

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Detalhes bibliográficos
Publicado no:Proc Natl Acad Sci U S A
Main Authors: Bogdanik, Laurent P., Osborne, Melissa A., Davis, Crystal, Martin, Whitney P., Austin, Andrew, Rigo, Frank, Bennett, C. Frank, Lutz, Cathleen M.
Formato: Artigo
Idioma:Inglês
Publicado em: National Academy of Sciences 2015
Assuntos:
PNAS Plus
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC4629342/
https://ncbi.nlm.nih.gov/pubmed/26460027
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1073/pnas.1509758112
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