AAV9-Stathmin1 gene delivery improves disease phenotype in an intermediate mouse model of spinal muscular atrophy

Spinal muscular atrophy (SMA) is a devastating infantile genetic disorder caused by the loss of survival motor neuron (SMN) protein that leads to premature death due to loss of motor neurons and muscle atrophy. The approval of an antisense oligonucleotide therapy for SMA was an important milestone i...

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Publicado en:Hum Mol Genet
Autores principales: Villalón, E, Kline, R A, Smith, C E, Lorson, Z C, Osman, E Y, O’Day, S, Murray, L M, Lorson, C L
Formato: Artigo
Lenguaje:Inglês
Publicado: Oxford University Press 2019
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Article
Acceso en línea:https://ncbi.nlm.nih.gov/pmc/articles/PMC6935388/
https://ncbi.nlm.nih.gov/pubmed/31363739
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1093/hmg/ddz188
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