A new biomarker candidate for spinal muscular atrophy: Identification of a peripheral blood cell population capable of monitoring the level of survival motor neuron protein

Spinal muscular atrophy (SMA) is a severe genetic neuromuscular disorder caused by insufficiency of functional survival motor neuron (SMN) protein. Several clinical trials have been conducted with the aim of upregulating the expression of the SMN protein in SMA patients. In order to evaluate the eff...

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Udgivet i:PLoS One
Main Authors: Otsuki, Noriko, Arakawa, Reiko, Kaneko, Kaori, Aoki, Ryoko, Arakawa, Masayuki, Saito, Kayoko
Format: Artigo
Sprog:Inglês
Udgivet: Public Library of Science 2018
Fag:
Research Article
Online adgang:https://ncbi.nlm.nih.gov/pmc/articles/PMC6089418/
https://ncbi.nlm.nih.gov/pubmed/30102724
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1371/journal.pone.0201764
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