Rescue of the spinal muscular atrophy phenotype in a mouse model by early postnatal delivery of SMN

Spinal muscular atrophy (SMA), the most common autosomal recessive neurodegenerative disease affecting children, results in impaired motor neuron function1. Despite knowledge of the pathogenic role of decreased survival motor neuron (SMN) protein levels, efforts to increase SMN have not resulted in...

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Bibliographische Detailangaben
Hauptverfasser: Foust, Kevin D, Wang, Xueyong, McGovern, Vicki L, Braun, Lyndsey, Bevan, Adam K, Haidet, Amanda M, Le, Thanh T, Morales, Pablo R, Rich, Mark M, Burghes, Arthur H M, Kaspar, Brian K
Format: Artigo
Sprache:Inglês
Veröffentlicht: 2010
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Article
Online Zugang:https://ncbi.nlm.nih.gov/pmc/articles/PMC2889698/
https://ncbi.nlm.nih.gov/pubmed/20190738
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/nbt.1610
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