Article
Dystrophin rescue by trans-splicing: a strategy for DMD genotypes not eligible for exon skipping approaches.
Nucleic acids research - 1 Sept 2013
Lorain Stéphanie, Peccate Cécile, Le Hir Maëva, Griffith Graziella, Philippi Susanne, Précigout Guillaume, Mamchaoui Kamel, Jollet Arnaud, Voit Thomas, Garcia Luis
Abstract excerpt
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 internal deletions, many mutations that affect nondispensable domains of the protein require further strategies. Among...
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