A multicenter comparison of quantification methods for antisense oligonucleotideinduced DMD exon 51 skipping in Duchenne muscular dystrophy cell cultures - Sorbonne Université
Journal Articles PLoS ONE Year : 2018

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

Ruurd C Verheul
  • Function : Author
Nicole A Datson
  • Function : Author
Pietro Spitali
  • Function : Author
  • PersonId : 1105152

Abstract

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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Dates and versions

hal-03285360 , version 1 (13-07-2021)

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Monika Hiller, Maria Sofia Falzarano, Iker Garcia-Jimenez, Valentina Sardone, Ruurd C Verheul, et al.. A multicenter comparison of quantification methods for antisense oligonucleotideinduced DMD exon 51 skipping in Duchenne muscular dystrophy cell cultures. PLoS ONE, 2018, 13 (10), pp.e0204485. ⟨10.1371/journal.pone.0204485⟩. ⟨hal-03285360⟩
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