Long-term dystrophin restoration supports development of splice correction therapy for DMD patients with exon 2 duplications
Author:
Publisher
Elsevier BV
Subject
Genetics,Molecular Biology,Molecular Medicine
Reference11 articles.
1. Therapeutic approaches for Duchenne muscular dystrophy;Roberts;Nat. Rev. Drug Discov.,2023
2. Duplications in the DMD gene;White;Hum. Mutat.,2006
3. Translation from a DMD exon 5 IRES results in a functional dystrophin isoform that attenuates dystrophinopathy in humans and mice;Wein;Nat. Med.,2014
4. DMD exon 1 truncating point mutations: amelioration of phenotype by alternative translation initiation in exon 6;Gurvich;Hum. Mutat.,2009
5. The first exon duplication mouse model of Duchenne muscular dystrophy: A tool for therapeutic development;Vulin;Neuromuscul. Disord.,2015
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