Motor neuron cell-nonautonomous rescue of spinal muscular atrophy phenotypes in mild and severe transgenic mouse models

Survival of motor neuron (SMN) deficiency causes spinal muscular atrophy (SMA), but the pathogenesis mechanisms remain elusive. Restoring SMN in motor neurons only partially rescues SMA in mouse models, although it is thought to be therapeutically essential. Here, we address the relative importance...

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Bibliographic Details
Published in:Genes Dev
Main Authors: Hua, Yimin, Liu, Ying Hsiu, Sahashi, Kentaro, Rigo, Frank, Bennett, C. Frank, Krainer, Adrian R.
Format: Artigo
Language:Inglês
Published: Cold Spring Harbor Laboratory Press 2015
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Research Paper
Online Access:https://ncbi.nlm.nih.gov/pmc/articles/PMC4318145/
https://ncbi.nlm.nih.gov/pubmed/25583329
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1101/gad.256644.114
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