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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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Bibliografiske detaljer
Udgivet i:PLoS One
Main Authors: 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.
Format: Artigo
Sprog:Inglês
Udgivet: Public Library of Science 2016
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Online adgang: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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