Second-generation compound for the modulation of utrophin in the therapy of DMD

Duchenne muscular dystrophy (DMD) is a lethal, X-linked muscle-wasting disease caused by lack of the cytoskeletal protein dystrophin. There is currently no cure for DMD although various promising approaches are progressing through human clinical trials. By pharmacologically modulating the expression...

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Detalhes bibliográficos
Publicado no:Hum Mol Genet
Main Authors: Guiraud, Simon, Squire, Sarah E., Edwards, Benjamin, Chen, Huijia, Burns, David T., Shah, Nandini, Babbs, Arran, Davies, Stephen G., Wynne, Graham M., Russell, Angela J., Elsey, David, Wilson, Francis X., Tinsley, Jon M., Davies, Kay E.
Formato: Artigo
Idioma:Inglês
Publicado em: Oxford University Press 2015
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Articles
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC4492389/
https://ncbi.nlm.nih.gov/pubmed/25935002
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1093/hmg/ddv154
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