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Skipping Multiple Exons to Treat DMD—Promises and Challenges

Duchenne muscular dystrophy (DMD) is a lethal disorder caused by mutations in the DMD gene. Antisense-mediated exon-skipping is a promising therapeutic strategy that makes use of synthetic nucleic acids to skip frame-disrupting exon(s) and allows for short but functional protein expression by restor...

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Bibliografiske detaljer
Udgivet i:Biomedicines
Main Authors: Aslesh, Tejal, Maruyama, Rika, Yokota, Toshifumi
Format: Artigo
Sprog:Inglês
Udgivet: MDPI 2018
Fag:
Online adgang:https://ncbi.nlm.nih.gov/pmc/articles/PMC5874658/
https://ncbi.nlm.nih.gov/pubmed/29301272
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.3390/biomedicines6010001
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