Nicolas Wein

Translation from a DMD exon 5 IRES results in a functional dystrophin isoform that attenuates dystrophinopathy in humans and mice

Nicolas Wein, Adeline Vulin, Maria Sofia Falzarano et al. · Nature Medicine · 2014 · 118 citations · Full text

Physiology, Ires Results, Degenerative Disease +3

Efficient bypass of mutations in dysferlin deficient patient cells by antisense-induced exon skipping

Nicolas Wein, Aurélie Avril, Marc Bartoli et al. · Human Mutation · 2009 · 88 citations · Full text

A Naturally Occurring Human Minidysferlin Protein Repairs Sarcolemmal Lesions in a Mouse Model of Dysferlinopathy

Martin Krahn, Nicolas Wein, Marc Bartoli et al. · Science Translational Medicine · 2010 · 84 citations · Full text

Clinical phenotypes as predictors of the outcome of skipping around <scp><i>DMD</i></scp> exon 45

Andrew R. Findlay, Nicolas Wein, Yuuki Kaminoh et al. · Annals of Neurology · 2015 · 50 citations · Full text

Intron mutations and early transcription termination in Duchenne and Becker muscular dystrophy

Megan A. Waldrop, Steven A. Moore, Katherine D. Mathews et al. · Human Mutation · 2022 · 47 citations · Full text