Normalization of Patient-Identified Plasma Biomarkers in SMNΔ7 Mice following Postnatal SMN Restoration

INTRODUCTION AND OBJECTIVE: Spinal muscular atrophy (SMA) is an autosomal recessive motor neuron disorder. SMA is caused by homozygous loss of the SMN1 gene and retention of the SMN2 gene resulting in reduced levels of full length SMN protein that are insufficient for motor neuron function. Various...

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Pubblicato in:PLoS One
Autori principali: Arnold, W. David, Duque, Sandra, Iyer, Chitra C., Zaworski, Phillip, McGovern, Vicki L., Taylor, Shannon J., von Herrmann, Katharine M., Kobayashi, Dione T., Chen, Karen S., Kolb, Stephen J., Paushkin, Sergey V., Burghes, Arthur H. M.
Natura: Artigo
Lingua:Inglês
Pubblicazione: Public Library of Science 2016
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Research Article
Accesso online:https://ncbi.nlm.nih.gov/pmc/articles/PMC5132001/
https://ncbi.nlm.nih.gov/pubmed/27907033
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1371/journal.pone.0167077
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