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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: | , , , , |
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| Formato: | Artigo |
| Idioma: | Inglês |
| Publicado em: |
Oxford University Press
2014
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| Assuntos: | |
| Acesso em linha: | 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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