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...

Volledige beschrijving

Bewaard in:
Bibliografische gegevens
Hoofdauteurs: Lorain, Stéphanie, Peccate, Cécile, Le Hir, Maëva, Griffith, Graziella, Philippi, Susanne, Précigout, Guillaume, Mamchaoui, Kamel, Jollet, Arnaud, Voit, Thomas, Garcia, Luis
Formaat: Artigo
Taal:Inglês
Gepubliceerd in: Oxford University Press 2013
Onderwerpen:
Synthetic Biology and Chemistry
Online toegang: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
Tags: Voeg label toe
Geen labels, Wees de eerste die dit record labelt!

Gelijkaardige items