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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Bibliografiska uppgifter
Huvudupphovsmän: Lorain, Stéphanie, Peccate, Cécile, Le Hir, Maëva, Griffith, Graziella, Philippi, Susanne, Précigout, Guillaume, Mamchaoui, Kamel, Jollet, Arnaud, Voit, Thomas, Garcia, Luis
Materialtyp: Artigo
Språk:Inglês
Publicerad: Oxford University Press 2013
Ämnen:
Synthetic Biology and Chemistry
Länkar: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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