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Myostatin inhibition in combination with antisense oligonucleotide therapy improves outcomes in spinal muscular atrophy

BACKGROUND: Spinal muscular atrophy (SMA) is caused by genetic defects in the survival motor neuron 1 (SMN1) gene that lead to SMN deficiency. Different SMN‐restoring therapies substantially prolong survival and function in transgenic mice of SMA. However, these therapies do not entirely prevent mus...

詳細記述

保存先:
書誌詳細
出版年:J Cachexia Sarcopenia Muscle
主要な著者: Zhou, Haiyan, Meng, Jinhong, Malerba, Alberto, Catapano, Francesco, Sintusek, Palittiya, Jarmin, Susan, Feng, Lucy, Lu‐Nguyen, Ngoc, Sun, Lianwen, Mariot, Virginie, Dumonceaux, Julie, Morgan, Jennifer E., Gissen, Paul, Dickson, George, Muntoni, Francesco
フォーマット: Artigo
言語:Inglês
出版事項: John Wiley and Sons Inc. 2020
主題:
オンライン・アクセス:https://ncbi.nlm.nih.gov/pmc/articles/PMC7296258/
https://ncbi.nlm.nih.gov/pubmed/32031328
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1002/jcsm.12542
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