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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Bibliografische gegevens
Gepubliceerd in:Hum Mol Genet
Hoofdauteurs: Villalón, E, Kline, R A, Smith, C E, Lorson, Z C, Osman, E Y, O’Day, S, Murray, L M, Lorson, C L
Formaat: Artigo
Taal:Inglês
Gepubliceerd in: Oxford University Press 2019
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Article
Online toegang: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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