How much dystrophin is enough: the physiological consequences of different levels of dystrophin in the mdx mouse

Splice modulation therapy has shown great clinical promise in Duchenne muscular dystrophy, resulting in the production of dystrophin protein. Despite this, the relationship between restoring dystrophin to established dystrophic muscle and its ability to induce clinically relevant changes in muscle f...

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Vydáno v:Hum Mol Genet
Hlavní autoři: Godfrey, Caroline, Muses, Sofia, McClorey, Graham, Wells, Kim E., Coursindel, Thibault, Terry, Rebecca L., Betts, Corinne, Hammond, Suzan, O'Donovan, Liz, Hildyard, John, El Andaloussi, Samir, Gait, Michael J., Wood, Matthew J., Wells, Dominic J.
Médium: Artigo
Jazyk:Inglês
Vydáno: Oxford University Press 2015
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Articles
On-line přístup:https://ncbi.nlm.nih.gov/pmc/articles/PMC4492390/
https://ncbi.nlm.nih.gov/pubmed/25935000
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1093/hmg/ddv155
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