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

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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

Edité par CCSD ; Public Library of Science -

International audience. 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 availability of standardized procedures to quantify exon skipping would be advantageous for the evaluation of preclinical and clinical data.

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