ArticleNature medicine2019
Non-immunogenic utrophin gene therapy for the treatment of muscular dystrophy animal models.
Open access · greenAbstract read
In one paragraph
Article in Nature medicine, 2019. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 64 papers, 2 of them syntheses that pooled it.
0numbers the graph read from it
0cells of the map it votes in
64citing papers in PubMed, 2 pooled it
4.7field-weighted citation impact, top 4% of its field
What it found
Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.
The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
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
64 citing papers in PubMed, 2 syntheses or guidelines pooled it, 86 citations in OpenAlex.
- Muscle and cardiac therapeutic strategies for Duchenne muscular dystrophy: past, present, and future.Pharmacological reports : PR · 2020Pooled it
- Recombinant Adeno-Associated Viral Vectors (rAAV)-Vector Elements in Ocular Gene Therapy Clinical Trials and Transgene Expression and Bioactivity Assays.International journal of molecular sciences · 2020Pooled it
- Duchenne muscular dystrophy: from gene to gene-ius therapies.Skeletal muscle · 2026Review
- CRISPR-Cas9-mediated upregulation of utrophin ameliorates Duchenne muscular dystrophy.Molecular therapy : the journal of the American Society of Gene Therapy · 2026Article
- Low-dose MyoAAV 2A-mediated delivery of engineered micro-utrophin achieves pan- muscle tissue distribution with elevated muscle function in Duchenne muscular dystrophy.Journal of translational medicine · 2026Article
- YAP Acts as a Negative Regulator of Mini Utrophin-Based Gene Therapy for Duchenne Muscular Dystrophy in Mdx Mice.International journal of molecular sciences · 2026Article
- RNA-Based Therapies for Treating Monogenic Cardiomyopathies.The Canadian journal of cardiology · 2026Review
- Dystrophins DP71 and DP427 determine cell viability during proliferation and myofibre differentiation.Cell death & disease · 2026Article
- Deaths in gene therapy of Duchenne muscular dystrophy and other diseases: Underlying mechanisms and mitigating strategies.Molecular therapy : the journal of the American Society of Gene Therapy · 2026Review
- 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
- (Z)-Endoxifen as a Potential Modulator of Utrophin Pathways in Duchenne Muscular Dystrophy: A Mechanistic and Transcriptomic Perspective.Degenerative neurological and neuromuscular disease · 2026Review
- Genetic strategies for therapy of Duchenne muscular dystrophy.Molecular therapy. Nucleic acids · 2025Review
- Systemic MyoAAV-saRNA delivery activates endogenous utrophin and rescues dystrophic pathology in mdx mice.Regenerative therapy · 2025Article
- Emerging Technologies Tackling Adeno-Associated Viruses (AAV) Immunogenicity in Gene Therapy Applications.Pharmaceutics · 2025Review
- Dystrophin Restorative and Compensatory Gene Addition Therapies for Duchenne Muscular Dystrophy: Could CRISPRa Provide a Realistic Alternative?Muscles (Basel, Switzerland) · 2025Review
- AAV-microutrophin gene therapy confers durable cardioprotection against pharmacologic and exercise-induced injury in the mdx mouse.Molecular therapy : the journal of the American Society of Gene Therapy · 2025Article
- Engineering Targeted Gene Delivery Systems for Primary Hereditary Skeletal Myopathies: Current Strategies and Future Perspectives.Biomedicines · 2025Review
- The road toward AAV-mediated gene therapy of Duchenne muscular dystrophy.Molecular therapy : the journal of the American Society of Gene Therapy · 2025Review
- Activation of endogenous full-length utrophin by MyoAAV-UA as a therapeutic approach for Duchenne muscular dystrophy.Nature communications · 2025Article
- Circulatory CCL2 distinguishes Duchenne muscular dystrophy dogs.Disease models & mechanisms · 2025Article
4 more citing papers are in PubMed but not listed here.
Corrections and comments
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Authors and funding
23 authors at 4 institutions in 1 country.
Yafeng Song *Department of Surgery, Pennsylvania Muscle Institute, Penn Cardiovascular Institute, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USA.
Leon Morales *Department of Surgery, Pennsylvania Muscle Institute, Penn Cardiovascular Institute, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USA.ORCID http://orcid.org/0000-0003-3079-8183
Alock S Malik *Department of Surgery, Pennsylvania Muscle Institute, Penn Cardiovascular Institute, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USA.
Andrew F MeadDepartment of Surgery, Pennsylvania Muscle Institute, Penn Cardiovascular Institute, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USA.
Christopher D GreerDepartment of Surgery, Pennsylvania Muscle Institute, Penn Cardiovascular Institute, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USA.ORCID http://orcid.org/0000-0003-2185-8500
Marilyn A MitchellDepartment of Surgery, Pennsylvania Muscle Institute, Penn Cardiovascular Institute, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USA.
Mihail T PetrovDepartment of Surgery, Pennsylvania Muscle Institute, Penn Cardiovascular Institute, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USA.
Leonard T SuDepartment of Surgery, Pennsylvania Muscle Institute, Penn Cardiovascular Institute, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USA.
Margaret E ChoiDepartment of Surgery, Pennsylvania Muscle Institute, Penn Cardiovascular Institute, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USA.
Shira T RosenblumDepartment of Surgery, Pennsylvania Muscle Institute, Penn Cardiovascular Institute, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USA.
Xiangping LuDepartment of Surgery, Pennsylvania Muscle Institute, Penn Cardiovascular Institute, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USA.
Daniel J VanBelzenDepartment of Surgery, Pennsylvania Muscle Institute, Penn Cardiovascular Institute, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USA.ORCID http://orcid.org/0000-0003-0949-3641
Ranjith K KrishnankuttyDepartment of Surgery, Pennsylvania Muscle Institute, Penn Cardiovascular Institute, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USA.
Frederick J BalzerDepartment of Surgery, Pennsylvania Muscle Institute, Penn Cardiovascular Institute, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USA.
Emanuele LoroDepartment of Physiology, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USA.
Robert FrenchDepartment of Surgery, Pennsylvania Muscle Institute, Penn Cardiovascular Institute, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USA.ORCID http://orcid.org/0000-0001-9914-4562
Kathleen J PropertDepartment of Biostatistics, Epidemiology and Informatics, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USA.
Shangzhen ZhouDepartment of Ophthalmology, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USA.
Benjamin W KozyakDepartment of Surgery, Pennsylvania Muscle Institute, Penn Cardiovascular Institute, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USA.
Peter P NghiemDepartment of Veterinary Integrative Biosciences, College of Veterinary Medicine and Texas A&M Institute for Neuroscience, Texas A&M University, College Station, TX, USA.
Tejvir S KhuranaDepartment of Physiology, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USA.
Joe N KornegayDepartment of Veterinary Integrative Biosciences, College of Veterinary Medicine and Texas A&M Institute for Neuroscience, Texas A&M University, College Station, TX, USA.
Hansell H StedmanDepartment of Surgery, Pennsylvania Muscle Institute, Penn Cardiovascular Institute, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USA. hstedman@pennmedicine.upenn.edu.ORCID http://orcid.org/0000-0003-2700-1815
University of Pennsylvania · USTexas A&M University · USChildren's Hospital of Philadelphia · USPhiladelphia VA Medical Center · US
Funding
Vector CoreP30DK047757 · NIDDK · UNIVERSITY OF PENNSYLVANIA · PI CALCEDO, ROBERTO · 1993 to 2017
$18.0MMultidisciplinary Training in Cardiovascular BiologyT32HL007954 · NHLBI · UNIVERSITY OF PENNSYLVANIA · PI DAVIES, PETER FRANCIS · 2000 to 2019
$9.9MThe Role of Hyaluronic Acid Receptors in Tendon HealingP30AR050950 · NIAMS · UNIVERSITY OF PENNSYLVANIA · PI PIGNOLO, ROBERT JOHN · 2006 to 2015
$6.2MTraining in Muscle Biology and Muscle DiseaseT32AR053461 · NIAMS · UNIVERSITY OF PENNSYLVANIA · PI OSTAP, E. MICHAEL · 2006 to 2025
$4.9MSystemic molecular therapy for muscular dystrophyR01NS042874 · NINDS · UNIVERSITY OF PENNSYLVANIA · PI STEDMAN, HANSELL H · 2002 to 2013
$4.8MNational Center for Canine Models of Duchenne Muscular Dystrophy (NCDMD)U24NS059696 · NINDS · UNIV OF NORTH CAROLINA CHAPEL HILL · PI KORNEGAY, JOE N. · 2008 to 2011
$2.1MTranslational program for molecular therapeutics in DMDU01NS052476 · NINDS · UNIVERSITY OF PENNSYLVANIA · PI STEDMAN, HANSELL H · 2007 to 2011
$2.0MShared Resource for Disease Model Surgical Critical Care and Data MangementS10RR028027 · NCRR · UNIVERSITY OF PENNSYLVANIA · PI STEDMAN, HANSELL H · 2010 to 2010
$481kNCRR NIH HHS S10 RR028027NHLBI NIH HHS T32 HL007954NIAMS NIH HHS P30 AR050950NIAMS NIH HHS T32 AR053461NIDDK NIH HHS P30 DK047757NINDS NIH HHS R01 NS042874NINDS NIH HHS U01 NS052476NINDS NIH HHS U24 NS059696
Abstract
The essential product of the Duchenne muscular dystrophy (DMD) gene is dystrophin
Indexed as
Genetic TherapyAnimalsDependovirusDisease Models, AnimalDogsDystrophinHumansMiceMice, Inbred mdxMuscle ContractionMuscle, SkeletalMuscular DystrophiesMuscular Dystrophy, AnimalMuscular Dystrophy, DuchenneTransgenesUtrophinDystrophinUtrophin
Identifiers
What OpenQuestion holds
Textmetadata
LicenceTDM
Read underepoch 390
Registered trials
None linked
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.