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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Udgivet i:Hum Mol Genet
Main Authors: Villalón, E, Kline, R A, Smith, C E, Lorson, Z C, Osman, E Y, O’Day, S, Murray, L M, Lorson, C L
Format: Artigo
Sprog:Inglês
Udgivet: Oxford University Press 2019
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Article
Online adgang: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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