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How do SMA-linked mutations of SMN1 lead to structural/functional deficiency of the SMA protein?

Spinal muscular atrophy (SMA) is an autosomal recessive neuromuscular disease with dysfunctional α-motor neurons in the anterior horn of the spinal cord. SMA is caused by loss (∼95% of SMA cases) or mutation (∼5% of SMA cases) of the survival motor neuron 1 gene SMN1. As the product of SMN1, SMN is...

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Bibliographische Detailangaben
Veröffentlicht in:PLoS One
1. Verfasser: Li, Wei
Format: Artigo
Sprache:Inglês
Veröffentlicht: Public Library of Science 2017
Schlagworte:
Online Zugang:https://ncbi.nlm.nih.gov/pmc/articles/PMC5453535/
https://ncbi.nlm.nih.gov/pubmed/28570645
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1371/journal.pone.0178519
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