A multicenter comparison of quantification methods for antisense oligonucleotide-induced DMD exon 51 skipping in Duchenne muscular dystrophy cell cultures

BACKGROUND: Duchenne muscular dystrophy is a lethal disease caused by lack of dystrophin. Skipping of exons adjacent to out-of-frame deletions has proven to restore dystrophin expression in Duchenne patients. Exon 51 has been the most studied target in both preclinical and clinical settings and the...

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Detalles Bibliográficos
Autores principales: Hiller, Monika, Falzarano, Maria Sofia, Garcia-Jimenez, Iker, Sardone, Valentina, Verheul, Ruurd C., Popplewell, Linda, Anthony, Karen, Ruiz-Del-Yerro, Estibaliz, Osman, Hana, Goeman, Jelle J., Mamchaoui, Kamel, Dickson, George, Ferlini, Alessandra, Muntoni, Francesco, Aartsma-Rus, Annemieke, Arechavala-Gomeza, Virginia, Datson, Nicole A., Spitali, Pietro
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Public Library of Science 2018
Materias:
Research Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6168132/
https://www.ncbi.nlm.nih.gov/pubmed/30278058
http://dx.doi.org/10.1371/journal.pone.0204485

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