Development of Exon Skipping Therapies for Duchenne Muscular Dystrophy: A Critical Review and a Perspective on the Outstanding Issues

Duchenne muscular dystrophy (DMD) is a rare, severe, progressive muscle-wasting disease leading to disability and premature death. Patients lack the muscle membrane-stabilizing protein dystrophin. Antisense oligonucleotide (AON)-mediated exon skipping is a therapeutic approach that aims to induce pr...

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Detalles Bibliográficos
Autores principales: Aartsma-Rus, Annemieke, Straub, Volker, Hemmings, Robert, Haas, Manuel, Schlosser-Weber, Gabriele, Stoyanova-Beninska, Violeta, Mercuri, Eugenio, Muntoni, Francesco, Sepodes, Bruno, Vroom, Elizabeth, Balabanov, Pavel
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Mary Ann Liebert, Inc. 2017
Materias:
Review
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5649120/
https://www.ncbi.nlm.nih.gov/pubmed/28796573
http://dx.doi.org/10.1089/nat.2017.0682

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