THE ANTISENSE TRANSCRIPT SMN-AS1 REGULATES SMN EXPRESSION AND IS A NOVEL THERAPEUTIC TARGET FOR SPINAL MUSCULAR ATROPHY

The neuromuscular disorder spinal muscular atrophy (SMA), the most common inherited killer of infants, is caused by insufficient expression of survival motor neuron (SMN) protein. SMA therapeutics development efforts have focused on identifying strategies to increase SMN expression. We identified a...

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Bibliografiske detaljer
Udgivet i:Neuron
Main Authors: d’Ydewalle, Constantin, Ramos, Daniel M., Pyles, Noah J., Ng, Shi Yan, Gorz, Mariusz, Pilato, Celeste M., Ling, Karen, Kong, Lingling, Ward, Amanda J., Rubin, Lee L., Rigo, Frank, Bennett, C. Frank, Sumner, Charlotte J.
Format: Artigo
Sprog:Inglês
Udgivet: 2016
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Article
Online adgang:https://ncbi.nlm.nih.gov/pmc/articles/PMC5223741/
https://ncbi.nlm.nih.gov/pubmed/28017471
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.neuron.2016.11.033
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