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Restoration of SMN to Emx-1 expressing cortical neurons is not sufficient to provide benefit to a severe mouse model of Spinal Muscular Atrophy

Spinal Muscular Atrophy (SMA), an autosomal recessive neuromuscular disorder, is a leading genetic cause of infant mortality. SMA is caused by the homozygous loss of Survival Motor Neuron-1 (SMN1). However, low, but essential, levels of SMN protein are produced by a nearly identical copy gene called...

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Wedi'i Gadw mewn:
Manylion Llyfryddiaeth
Prif Awduron: Taylor, Alexander S., Glascock, Jacqueline J., Rose, Ferrill F., Lutz, Cathleen, Lorson, Christian L.
Fformat: Artigo
Iaith:Inglês
Cyhoeddwyd: 2013
Pynciau:
Mynediad Ar-lein:https://ncbi.nlm.nih.gov/pmc/articles/PMC3745810/
https://ncbi.nlm.nih.gov/pubmed/23512182
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1007/s11248-013-9702-y
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