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References from U7snRNA-mediated skipping of intron-derived pseudoexons restores full-length DMD expression in patient-derived cell lines. Local targets link to admitted publications; unresolved targets remain external evidence.
the protein product of the Duchenne/Becker muscular dystrophy gene
10.1016/0968-0004(89)90290-9 · 1989 · External reference
The cell biology of disease: cellular and molecular mechanisms underlying muscular dystrophy
10.1083/jcb.201212142 · 2013 · External reference
Life Expectancy in Duchenne Muscular Dystrophy: Reproduced Individual Patient Data Meta-analysis
10.1212/wnl.0000000000012910 · 2021 · External reference
Increase in Full-Length Dystrophin by Exon Skipping in Duchenne Muscular Dystrophy Patients with Single Exon Duplications: An Open-label Study
10.3233/jnd-230107 · 2024 · External reference
Longitudinal effect of eteplirsen versus historical control on ambulation in Duchenne muscular dystrophy
10.1002/ana.24555 · 2016 · External reference
Clinical applications of exon-skipping antisense oligonucleotides in neuromuscular diseases
10.1016/j.ymthe.2025.04.038 · 2025 · External reference
Unresolved reference
2023 · External reference
AAV gene therapy for Duchenne muscular dystrophy: the EMBARK phase 3 randomized trial
10.1038/s41591-024-03304-z · 2025 · External reference
Microdystrophin Gene Therapy for Duchenne Muscular Dystrophy: The Ethics and Financial Toxicity of Exuberant Hope
10.1212/wnl.0000000000214085 · 2025 · External reference
Assembly, nuclear import and function of U7 snRNPs studied by microinjection of synthetic U7 RNA into Xenopus oocytes
10.1093/nar/23.16.3141 · 1995 · External reference
Stable alteration of pre-mRNA splicing patterns by modified U7 small nuclear RNAs
10.1073/pnas.95.9.4929 · 1998 · External reference
U7 snRNA, a Small RNA with a Big Impact in Gene Therapy
2021 · External reference
Rescue of dystrophic muscle through U7 snRNA-mediated exon skipping
10.1126/science.1104297 · 2004 · External reference
U7 snRNAs induce correction of mutated dystrophin pre-mRNA by exon skipping
10.1007/s000180300047 · 2003 · External reference
U7 snRNA: A tool for gene therapy
10.1002/jgm.3321 · 2021 · External reference
Pseudoexons of the DMD Gene
10.3233/jnd-190431 · 2020 · External reference
Improved molecular diagnosis of dystrophinopathies in an unselected clinical cohort
10.1002/ajmg.a.30617 · 2005 · External reference
Intron mutations and early transcription termination in Duchenne and Becker muscular dystrophy
10.1002/humu.24343 · 2022 · External reference
Direct Reprogramming of Human Fibroblasts into Myoblasts to Investigate Therapies for Neuromuscular Disorders
2021 · External reference
Eteplirsen for the treatment of Duchenne muscular dystrophy
10.1002/ana.23982 · 2013 · External reference
Viltolarsen in Japanese Duchenne muscular dystrophy patients: A phase 1/2 study
10.1002/acn3.51235 · 2020 · External reference
Factor IX expression in skeletal muscle of a severe hemophilia B patient 10 years after AAV-mediated gene transfer
10.1182/blood-2011-09-382317 · 2012 · External reference
Durability of transgene expression after rAAV gene therapy
10.1016/j.ymthe.2022.03.004 · 2022 · External reference
Persistence of exon 2 skipping and dystrophin expression at 18 months after U7snRNA-mediated therapy in the Dup2 mouse model
10.1016/j.omtm.2023.101144 · 2023 · External reference
Lack of Toxicity in Nonhuman Primates Receiving Clinically Relevant Doses of an AAV9.U7snRNA Vector Designed to Induce DMD Exon 2 Skipping
10.1089/hum.2020.286 · 2021 · External reference
Pre-clinical dose-escalation studies establish a therapeutic range for U7snRNA-mediated DMD exon 2 skipping
10.1016/j.omtm.2021.03.014 · 2021 · External reference
A survey of ex vivo/in vitro transduction efficiency of mammalian primary cells and cell lines with Nine natural adeno-associated virus (AAV1-9) and one engineered adeno-associated virus serotype
10.1186/1743-422x-10-74 · 2013 · External reference
AOC 1044 induces exon 44 skipping and restores dystrophin protein in preclinical models of Duchenne muscular dystrophy
10.1093/nar/gkaf241 · 2025 · External reference