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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Bibliographische Detailangaben
Hauptverfasser: Lorain, Stéphanie, Peccate, Cécile, Le Hir, Maëva, Griffith, Graziella, Philippi, Susanne, Précigout, Guillaume, Mamchaoui, Kamel, Jollet, Arnaud, Voit, Thomas, Garcia, Luis
Format: Artigo
Sprache:Inglês
Veröffentlicht: Oxford University Press 2013
Schlagworte:
Synthetic Biology and Chemistry
Online Zugang: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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