Evaluation of 2'-Deoxy-2'-fluoro Antisense Oligonucleotides for Exon Skipping in Duchenne Muscular Dystrophy

Duchenne muscular dystrophy (DMD) is a severe muscle wasting disorder typically caused by frame-shifting mutations in the DMD gene. Restoration of the reading frame would allow the production of a shorter but partly functional dystrophin protein as seen in Becker muscular dystrophy patients. This ca...

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Detalles Bibliográficos
Autores principales: Jirka, Silvana M G, Tanganyika-de Winter, Christa L, Boertje-van der Meulen, Joke W, van Putten, Maaike, Hiller, Monika, Vermue, Rick, de Visser, Peter C, Aartsma-Rus, Annemieke
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Nature Publishing Group 2015
Materias:
Original Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5014533/
https://www.ncbi.nlm.nih.gov/pubmed/26623937
http://dx.doi.org/10.1038/mtna.2015.39

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5014533/
https://www.ncbi.nlm.nih.gov/pubmed/26623937
http://dx.doi.org/10.1038/mtna.2015.39

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