The potential of utrophin and dystrophin combination therapies for Duchenne muscular dystrophy

Duchenne muscular dystrophy (DMD) is a lethal neuromuscular disorder caused by loss of dystrophin. Several therapeutic modalities are currently in clinical trials but none will achieve maximum functional rescue and full disease correction. Therefore, we explored the potential of combining the benefi...

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Dettagli Bibliografici
Pubblicato in:Hum Mol Genet
Autori principali: Guiraud, Simon, Edwards, Benjamin, Babbs, Arran, Squire, Sarah E, Berg, Adam, Moir, Lee, Wood, Matthew J, Davies, Kay E
Natura: Artigo
Lingua:Inglês
Pubblicazione: Oxford University Press 2019
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General Article
Accesso online:https://ncbi.nlm.nih.gov/pmc/articles/PMC6586144/
https://ncbi.nlm.nih.gov/pubmed/30990876
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1093/hmg/ddz049
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