ArticleHuman gene therapy2023
Microdystrophin Expression as a Surrogate Endpoint for Duchenne Muscular Dystrophy Clinical Trials.
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In one paragraph
Article in Human gene therapy, 2023. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 25 papers.
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0cells of the map it votes in
25citing papers in PubMed
4.0field-weighted citation impact, top 6% of its field
What it found
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The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.
Who cites it
25 citing papers in PubMed, 26 citations in OpenAlex.
- The Multi-System Roles of Dp71 Dystrophin Isoforms in Duchenne Muscular Dystrophy.Muscles (Basel, Switzerland) · 2026Review
- Adeno-Associated Virus Vector Mediated Gene Therapy: A Promising Approach to Transform Hypertrophic Cardiomyopathy Treatment.Biotechnology journal · 2026Review
- Quantification of a serum titin fragment reflects dystrophin restoration in mdx mice and disease severity in patients with dystrophinopathies.Journal of neuromuscular diseases · 2026Article
- Gene Editing Strategies for Duchenne Muscular Dystrophy: From Molecular Mechanisms to Clinical Translation.Cells · 2026Review
- Current and Emerging Therapeutic Strategies for the Treatment of Duchenne Muscular Dystrophy.Genes · 2026Review
- Quantitative Measurement of Glycosylated ⍺-Dystroglycan as a Biomarker for Disease Severity in Limb-Girdle Muscular Dystrophy Type 2I/R9.Neurology. Genetics · 2026Article
- Current Trends in Duchenne Muscular Dystrophy Research and Therapy: 3D Cardiac Modelling.Journal of cachexia, sarcopenia and muscle · 2026Review
- Molecular Bases of Myopathies and Their Impact on Clinical Practice: Advances and Future Perspectives.International journal of molecular sciences · 2026Review
- Dystrophin/mini-dystrophin expression analysis by immunoaffinity liquid chromatography-tandem mass spectrometry after gene therapy for DMD.Gene therapy · 2025Article
- Emerging Technologies Tackling Adeno-Associated Viruses (AAV) Immunogenicity in Gene Therapy Applications.Pharmaceutics · 2025Review
- Lysosomal damage is a therapeutic target in Duchenne muscular dystrophy.Science advances · 2025Article
- Synthetic bottlebrush block copolymer prevents disease onset in Duchenne muscular dystrophy.Proceedings of the National Academy of Sciences of the United States of America · 2025Article
- Large-scale serum protein biomarkers discovery associated with function and clinical milestones in Duchenne muscular dystrophy.Nature communications · 2025Observational
- AAV microdystrophin gene replacement therapy for Duchenne muscular dystrophy: progress and prospects.Gene therapy · 2025Review
- Contrasting Becker and Duchenne muscular dystrophy serum biomarker candidates by using data independent acquisition LC-MS/MS.Skeletal muscle · 2025Article
- Next-generation replication-defective HSV vectors for delivery of large DNA payloads.Molecular therapy : the journal of the American Society of Gene Therapy · 2025Review
- Unlocking the Mysteries of Rare Disease Drug Development: A Beginner's Guide for Clinical Pharmacologists.Clinical and translational science · 2025Review
- "If you cannot measure it, you cannot improve it". Outcome measures in Duchenne Muscular Dystrophy: current and future perspectives.Acta neurologica Belgica · 2025Review
- Aptamer-conjugated gold nanoparticles enable oligonucleotide delivery into muscle stem cells to promote regeneration of dystrophic muscles.Nature communications · 2025Article
- A Hypothesized Therapeutic Role of (Z)-Endoxifen in Duchenne Muscular Dystrophy (DMD).Degenerative neurological and neuromuscular disease · 2025Article
Corrections and comments
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Authors and funding
14 authors at 8 institutions in 3 countries.
Jeffrey S ChamberlainDepartment of Neurology, Sen. Paul D. Wellstone Muscular Dystrophy Specialized Research Center, University of Washington, Seattle, Washington, USA.
Melissa RobbRobb Consulting (contractor of REGENXBIO, Inc.), Columbia, Maryland, USA.ORCID 0000-0001-7924-8604
Serge BraunAFMTelethon, Evry, France.
Kristy J BrownSolid Biosciences, Inc., Charlestown, Massachusetts, USA.
Olivier DanosREGENXBIO, Inc., Rockville, Maryland, USA.
Annie GanotSolid Biosciences, Inc., Charlestown, Massachusetts, USA.
Pedro Gonzalez-AlegreSpark Therapeutics, Inc., Philadelphia, Pennsylvania, USA.
Nina HunterREGENXBIO, Inc., Rockville, Maryland, USA.
Craig McDonaldUniversity of California Davis Medical Center, Sacramento, California, USA.
Carl MorrisSolid Biosciences, Inc., Charlestown, Massachusetts, USA.
Mark TobolowskyHyman, Phelps & McNamara, P.C. (former contractor of REGENXBIO, Inc.), Washington, District of Columbia, USA.
Kathryn R WagnerF. Hoffmann-La Roche, Ltd., Basel, Switzerland.
Olivia ZiolkowskiFormer contractor of REGENXBIO, Inc., Rockville, Maryland, USA; and.
Dongsheng DuanDepartment of Molecular Microbiology and Immunology, Neurology, Biomedical Sciences, and Biomedical, Biological & Chemical Engineering, University of Missouri, Columbia, Missouri, USA.
Regenxbio (United States) · USSolidus Biosciences (United States) · USAssociation Francaise contre les Myopathies · FRRoche (Switzerland) · CHSpark Therapeutics (United States) · USUniversity of California Davis Medical Center · USUniversity of Missouri · USUniversity of Washington · US
Funding
Vector and Transgenic Mouse CoreP30DK017047 · NIDDK · UNIVERSITY OF WASHINGTON · PI Karin E Bornfeldt · 1986 to 2026
$41.4MDYSTROPHIN REPLACEMENT IN MDX MICER01AR040864 · NIAMS · UNIVERSITY OF WASHINGTON · PI JEFFREY S CHAMBERLAIN · 1991 to 2026
$8.8MWhole body single AAV microgene therapy in canine DMDR01NS090634 · NINDS · UNIVERSITY OF MISSOURI-COLUMBIA · PI Dongsheng Duan · 2015 to 2026
$4.2MOptimizing and validation of gene therapy vectors to treat limb girdle muscular dystophyR01NS117912 · NINDS · UNIVERSITY OF CALIFORNIA LOS ANGELES · PI CHAMBERLAIN, JEFFREY S, SPENCER, MELISSA JAN · 2020 to 2024
$2.6MNIAMS NIH HHS R01 AR040864NIDDK NIH HHS P30 DK017047NINDS NIH HHS R01 NS090634NINDS NIH HHS R01 NS117912
Abstract
Duchenne muscular dystrophy (DMD) is a serious, rare genetic disease, affecting primarily boys. It is caused by mutations in the
Indexed as
Muscular Dystrophy, DuchenneBiomarkersGenetic TherapyGene Transfer TechniquesHumansMaleMuscle, SkeletalBiomarkersAAV vectorDuchenne muscular dystrophygene therapymicrodystrophin
Identifiers
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LicenceCC BY
Read underepoch 390
Registered trials
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Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.