ArticleMolecular therapy. Methods & clinical development2022
A first-in-human phase I/IIa gene transfer clinical trial for Duchenne muscular dystrophy using rAAVrh74.MCK.
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Article in Molecular therapy. Methods & clinical development, 2022. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 21 papers, 2 of them syntheses that pooled it.
0numbers the graph read from it
0cells of the map it votes in
21citing papers in PubMed, 2 pooled it
2.0field-weighted citation impact, top 13% 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
21 citing papers in PubMed, 2 syntheses or guidelines pooled it, 31 citations in OpenAlex.
- Incidence, timing, and clinical significance of adverse immune events after gene replacement therapy: A systematic review and meta-analysis.Molecular therapy : the journal of the American Society of Gene Therapy · 2026Pooled it
- A systematic review of immunosuppressive protocols used in AAV gene therapy for monogenic disorders.Molecular therapy : the journal of the American Society of Gene Therapy · 2024Pooled it
- Analysis of Dystrophin-Associated Glycoproteins: Focus on Expression and Glycosylation of Dystroglycan in Skeletal Muscle.Methods in molecular biology (Clifton, N.J.) · 2026Article
- Automated Quantification of Dystrophin Expression by Immunofluorescence in Humans and Animal Models.Methods in molecular biology (Clifton, N.J.) · 2026Article
- From molecular correction to functional rescue: delivery, tissue state, and immunobiology as the principal constraints on dystrophin-restoring therapy in Duchenne muscular dystrophy.Frontiers in pharmacology · 2026Review
- Genetic strategies for therapy of Duchenne muscular dystrophy.Molecular therapy. Nucleic acids · 2025Review
- Dystrophin Restorative and Compensatory Gene Addition Therapies for Duchenne Muscular Dystrophy: Could CRISPRa Provide a Realistic Alternative?Muscles (Basel, Switzerland) · 2025Review
- Review
- Continuous adipose-derived stem cell therapy from the neonatal stage effectively reduces Duchenne muscular dystrophy symptoms in rats.Stem cell research & therapy · 2025Article
- Fully personalized modelling of Duchenne Muscular Dystrophy ambulation.Philosophical transactions. Series A, Mathematical, physical, and engineering sciences · 2025Article
- Dual FKRP/FST gene therapy normalizes ambulation, increases strength, decreases pathology, and amplifies gene expression in LGMDR9 mice.Molecular therapy : the journal of the American Society of Gene Therapy · 2024Article
- Adeno-associated virus as a delivery vector for gene therapy of human diseases.Signal transduction and targeted therapy · 2024Review
- Interspecies Scaling of Transgene Products for Viral Vector Gene Therapies: Method Assessment Using Data from Eleven Viral Vectors.The AAPS journal · 2023Article
- Step Activity Monitoring in Boys with Duchenne Muscular Dystrophy and its Correlation with Magnetic Resonance Measures and Functional Performance.Journal of neuromuscular diseases · 2022Article
- Genome-wide activation screens to increase adeno-associated virus production.Molecular therapy. Nucleic acids · 2021Article
- Evaluating the potential of novel genetic approaches for the treatment of Duchenne muscular dystrophy.European journal of human genetics : EJHG · 2021Review
- Mechanistic model for production of recombinant adeno-associated virus via triple transfection of HEK293 cells.Molecular therapy. Methods & clinical development · 2021Article
- B4GALNT2 Gene Promotes Proliferation, and Invasiveness and Migration Abilities of Model Triple Negative Breast Cancer (TNBC) Cells by Interacting With HLA-B Protein.Frontiers in oncology · 2021Article
- T Cell-Mediated Immune Responses to AAV and AAV Vectors.Frontiers in immunology · 2021Review
- Gene Therapy for Heart Failure: New Perspectives.Current heart failure reports · 2018Review
Corrections and comments
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Authors and funding
15 authors at 2 institutions in 1 country.
Kevin M FlaniganCenter for Gene Therapy, Abigail Wexner Research Institute, Nationwide Children's Hospital, 700 Children's Drive, Columbus, OH 43205, USA.
Tatyana A VetterCenter for Gene Therapy, Abigail Wexner Research Institute, Nationwide Children's Hospital, 700 Children's Drive, Columbus, OH 43205, USA.
Tabatha R SimmonsCenter for Gene Therapy, Abigail Wexner Research Institute, Nationwide Children's Hospital, 700 Children's Drive, Columbus, OH 43205, USA.
Megan IammarinoCenter for Gene Therapy, Abigail Wexner Research Institute, Nationwide Children's Hospital, 700 Children's Drive, Columbus, OH 43205, USA.
Emma C FrairCenter for Gene Therapy, Abigail Wexner Research Institute, Nationwide Children's Hospital, 700 Children's Drive, Columbus, OH 43205, USA.
Federica RinaldiCenter for Gene Therapy, Abigail Wexner Research Institute, Nationwide Children's Hospital, 700 Children's Drive, Columbus, OH 43205, USA.
Louis G ChicoineCenter for Gene Therapy, Abigail Wexner Research Institute, Nationwide Children's Hospital, 700 Children's Drive, Columbus, OH 43205, USA.
Johan HarrisCenter for Gene Therapy, Abigail Wexner Research Institute, Nationwide Children's Hospital, 700 Children's Drive, Columbus, OH 43205, USA.
John P CheathamDepartment of Pediatrics, The Ohio State University College of Medicine, Columbus, OH, USA.
Sharon L CheathamDepartment of Pediatrics, The Ohio State University College of Medicine, Columbus, OH, USA.
Brian BoeDepartment of Pediatrics, The Ohio State University College of Medicine, Columbus, OH, USA.
Megan A WaldropCenter for Gene Therapy, Abigail Wexner Research Institute, Nationwide Children's Hospital, 700 Children's Drive, Columbus, OH 43205, USA.
Deborah A ZygmuntCenter for Gene Therapy, Abigail Wexner Research Institute, Nationwide Children's Hospital, 700 Children's Drive, Columbus, OH 43205, USA.
Davin PackerNeuroscience Graduate Program, The Ohio State University, Columbus, OH, USA.
Paul T MartinCenter for Gene Therapy, Abigail Wexner Research Institute, Nationwide Children's Hospital, 700 Children's Drive, Columbus, OH 43205, USA.
Nationwide Children's Hospital · USThe Ohio State University · US
Funding
Project 3: Use of an IRES-driven N-truncated dystrophin isoform as a clinical therapy for 5 mutations in the dystrophinopathiesP50AR070604 · NIAMS · RESEARCH INST NATIONWIDE CHILDREN'S HOSP · PI MCBRIDE, KIM LEWIS · 2016 to 2020
$7.3MFirst-in-Human rAAVrh74.MCK.GALGT2 DMD Clinical TrialR21AR068040 · NIAMS · RESEARCH INST NATIONWIDE CHILDREN'S HOSP · PI FLANIGAN, KEVIN M · 2015 to 2017
$526kNIAMS NIH HHS P50 AR070604NIAMS NIH HHS R21 AR068040
Abstract
In a phase 1/2, open-label dose escalation trial, we delivered rAAVrh74.MCK.
Indexed as
AAVB4GALNTDuchenne muscular dystrophyGALGT2gene therapy
Identifiers
What OpenQuestion holds
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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.