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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Publicat a:Neuron
Autors principals: 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
Idioma:Inglês
Publicat: 2016
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Article
Accés en línia: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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