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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Detalles Bibliográficos
Autores principales: Aslesh, Tejal, Maruyama, Rika, Yokota, Toshifumi
Formato: Online Artículo Texto
Lenguaje:English
Publicado: MDPI 2018
Materias:
Review
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5874658/
https://www.ncbi.nlm.nih.gov/pubmed/29301272
http://dx.doi.org/10.3390/biomedicines6010001

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5874658/
https://www.ncbi.nlm.nih.gov/pubmed/29301272
http://dx.doi.org/10.3390/biomedicines6010001

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