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...

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Detalhes bibliográficos
Publicado no:J Cachexia Sarcopenia Muscle
Main Authors: 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
Formato: Artigo
Idioma:Inglês
Publicado em: John Wiley and Sons Inc. 2020
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Original Articles
Acesso em linha: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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