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Dystrophin-deficient large animal models: translational research and exon skipping

Duchenne muscular dystrophy (DMD) is an X-linked recessive genetic disorder caused by mutations in the dystrophin gene. Affecting approximately 1 in 3,600-9337 boys, DMD patients exhibit progressive muscle degeneration leading to fatality as a result of heart or respiratory failure. Despite the seve...

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Dettagli Bibliografici
Pubblicato in:Am J Transl Res
Autori principali: Yu, Xinran, Bao, Bo, Echigoya, Yusuke, Yokota, Toshifumi
Natura: Artigo
Lingua:Inglês
Pubblicazione: e-Century Publishing Corporation 2015
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Accesso online:https://ncbi.nlm.nih.gov/pmc/articles/PMC4568789/
https://ncbi.nlm.nih.gov/pubmed/26396664
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