Dystrophin rescue by trans-splicing: a strategy for DMD genotypes not eligible for exon skipping approaches

RNA-based therapeutic approaches using splice-switching oligonucleotides have been successfully applied to rescue dystrophin in Duchenne muscular dystrophy (DMD) preclinical models and are currently being evaluated in DMD patients. Although the modular structure of dystrophin protein tolerates inter...

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Detalles Bibliográficos
Autores principales: Lorain, Stéphanie, Peccate, Cécile, Le Hir, Maëva, Griffith, Graziella, Philippi, Susanne, Précigout, Guillaume, Mamchaoui, Kamel, Jollet, Arnaud, Voit, Thomas, Garcia, Luis
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Oxford University Press 2013
Materias:
Synthetic Biology and Chemistry
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC3783188/
https://www.ncbi.nlm.nih.gov/pubmed/23861443
http://dx.doi.org/10.1093/nar/gkt621

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC3783188/
https://www.ncbi.nlm.nih.gov/pubmed/23861443
http://dx.doi.org/10.1093/nar/gkt621

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