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Translation from a DMD exon 5 IRES results in a functional dystrophin isoform that attenuates dystrophinopathy in humans and mice.
Wein N, Vulin A, Falzarano MS, Szigyarto CA, Maiti B, Findlay A, Heller KN, Uhlén M, Bakthavachalu B, Messina S, Vita G, Passarelli C, Brioschi S, Bovolenta M, Neri M, Gualandi F, Wilton SD, Rodino-Klapac LR, Yang L, Dunn DM, Schoenberg DR, Weiss RB, Howard MT, Ferlini A, Flanigan KM. Wein N, et al. Among authors: wilton sd. Nat Med. 2014 Sep;20(9):992-1000. doi: 10.1038/nm.3628. Epub 2014 Aug 10. Nat Med. 2014. PMID: 25108525 Free PMC article.
Antisense-induced exon skipping and synthesis of dystrophin in the mdx mouse.
Mann CJ, Honeyman K, Cheng AJ, Ly T, Lloyd F, Fletcher S, Morgan JE, Partridge TA, Wilton SD. Mann CJ, et al. Among authors: wilton sd. Proc Natl Acad Sci U S A. 2001 Jan 2;98(1):42-7. doi: 10.1073/pnas.98.1.42. Proc Natl Acad Sci U S A. 2001. PMID: 11120883 Free PMC article.
Induced dystrophin exon skipping in human muscle explants.
McClorey G, Fall AM, Moulton HM, Iversen PL, Rasko JE, Ryan M, Fletcher S, Wilton SD. McClorey G, et al. Among authors: wilton sd. Neuromuscul Disord. 2006 Oct;16(9-10):583-90. doi: 10.1016/j.nmd.2006.05.017. Epub 2006 Aug 21. Neuromuscul Disord. 2006. PMID: 16919955
207 results