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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Publicat a:Hum Mol Genet
Autors principals: 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.
Format: Artigo
Idioma:Inglês
Publicat: Oxford University Press 2015
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Articles
Accés en línia: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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