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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Udgivet i: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.
Format: Artigo
Sprog:Inglês
Udgivet: Oxford University Press 2015
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Articles
Online adgang: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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