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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Main Authors: Lorain, Stéphanie, Peccate, Cécile, Le Hir, Maëva, Griffith, Graziella, Philippi, Susanne, Précigout, Guillaume, Mamchaoui, Kamel, Jollet, Arnaud, Voit, Thomas, Garcia, Luis
Format: Artigo
Sprog:Inglês
Udgivet: Oxford University Press 2013
Fag:
Synthetic Biology and Chemistry
Online adgang:https://ncbi.nlm.nih.gov/pmc/articles/PMC3783188/
https://ncbi.nlm.nih.gov/pubmed/23861443
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1093/nar/gkt621
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