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Investigation of New Morpholino Oligomers to Increase Survival Motor Neuron Protein Levels in Spinal Muscular Atrophy

Spinal muscular atrophy (SMA) is an autosomal-recessive childhood motor neuron disease and the main genetic cause of infant mortality. SMA is caused by deletions or mutations in the survival motor neuron 1 (SMN1) gene, which results in SMN protein deficiency. Only one approved drug has recently beco...

詳細記述

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書誌詳細
主要な著者: Agnese Ramirez, Sebastiano G. Crisafulli, Mafalda Rizzuti, Nereo Bresolin, Giacomo P. Comi, Stefania Corti, Monica Nizzardo
フォーマット: Artigo
言語:Inglês
出版事項: MDPI AG 2018-01-01
シリーズ:International Journal of Molecular Sciences
主題:
オンライン・アクセス:http://www.mdpi.com/1422-0067/19/1/167
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