Long-Term Efficacy of Systemic Multiexon Skipping Targeting Dystrophin Exons 45–55 With a Cocktail of Vivo-Morpholinos in Mdx52 Mice

Antisense-mediated exon skipping, which can restore the reading frame, is a most promising therapeutic approach for Duchenne muscular dystrophy. Remaining challenges include the limited applicability to patients and unclear function of truncated dystrophin proteins. Multiexon skipping targeting exon...

Descripción completa

Detalles Bibliográficos
Autores principales: Echigoya, Yusuke, Aoki, Yoshitsugu, Miskew, Bailey, Panesar, Dharminder, Touznik, Aleksander, Nagata, Tetsuya, Tanihata, Jun, Nakamura, Akinori, Nagaraju, Kanneboyina, Yokota, Toshifumi
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/PMC4345310/
https://www.ncbi.nlm.nih.gov/pubmed/25647512
http://dx.doi.org/10.1038/mtna.2014.76

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4345310/
https://www.ncbi.nlm.nih.gov/pubmed/25647512
http://dx.doi.org/10.1038/mtna.2014.76

Ejemplares similares