ArticleScience translational medicine2023
Assessment of systemic AAV-microdystrophin gene therapy in the GRMD model of Duchenne muscular dystrophy.
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Article in Science translational medicine, 2023. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 48 papers.
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48citing papers in PubMed
10.0field-weighted citation impact, top 1% of its field
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Who cites it
48 citing papers in PubMed, 65 citations in OpenAlex.
- Decoding Nonlinearities in AAV-Based Gene Therapy Using PBPK Modelling.The AAPS journal · 2026Article
- AAV-mediated gene transfer of a novel microdystrophin ameliorates pathology and enhances muscle function in a mouse model of DMD.Molecular therapy. Nucleic acids · 2026Article
- AAVcc47-The Yale journal of biology and medicine · 2026Article
- 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
- Preclinical evaluation of INS1201 AAV9-micro-dystrophin via CSF administration as a potential therapy for Duchenne muscular dystrophy.Molecular therapy. Advances · 2026Article
- The D2.B10-DmdThe American journal of pathology · 2026Article
- Proteomics-based evaluation of AAV dystrophin gene therapy outcomes in mdx skeletal muscle.JCI insight · 2026Article
- Dose- and genotype-dependent cardiac arrhythmia and sudden death in rats following microdystrophin gene therapy.Molecular therapy : the journal of the American Society of Gene Therapy · 2026Article
- Canine Model of Duchenne Muscular Dystrophy.Methods in molecular biology (Clifton, N.J.) · 2026Article
- Manual Immunofluorescence Staining for Dystrophin and Key Dystrophin-Associated Proteins.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
- AAV microdystrophin gene replacement therapy for Duchenne muscular dystrophy: progress and prospects.Gene therapy · 2025Review
- Lymphatic dysfunction is linked to disease pathogenesis in Duchenne muscular dystrophy animal models.Proceedings of the National Academy of Sciences of the United States of America · 2025Article
- Enhanced therapeutic potential of a microdystrophin with an extended C-terminal domain.Molecular therapy. Methods & clinical development · 2025Article
- Long-Term Dystrophin Replacement Therapy in Duchenne Muscular Dystrophy Causes Cardiac Inflammation.JACC. Basic to translational science · 2025Article
- The road toward AAV-mediated gene therapy of Duchenne muscular dystrophy.Molecular therapy : the journal of the American Society of Gene Therapy · 2025Review
- Circulatory CCL2 distinguishes Duchenne muscular dystrophy dogs.Disease models & mechanisms · 2025Article
- Beta-hydroxy-beta-methylbutyrate (HMB) improves daily activity and whole-body protein metabolism in Duchenne muscular dystrophy dogs: a pilot study.Scientific reports · 2025Article
Corrections and comments
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Authors and funding
31 authors at 9 institutions in 1 country.
Sharla M BirchTexas A&M University, College of Veterinary Medicine and Biomedical Sciences, College Station, TX 77843, USA.ORCID 0000-0002-8088-8104
Michael W LawlorMedical College of Wisconsin, Milwaukee, WI 53226, USA.ORCID 0000-0003-0731-3950
Thomas J ConlonPowell Gene Therapy Center, University of Florida, Gainesville, FL 32610, USA.
Lee-Jae GuoTexas A&M University, College of Veterinary Medicine and Biomedical Sciences, College Station, TX 77843, USA.ORCID 0000-0002-9633-1167
Julie M CrudeleUniversity of Washington, Seattle, WA 98109, USA.ORCID 0000-0003-4472-0125
Eleanor C HawkinsCollege of Veterinary Medicine, North Carolina State University, Raleigh, NC 27606, USA.ORCID 0000-0002-4006-184X
Peter P NghiemTexas A&M University, College of Veterinary Medicine and Biomedical Sciences, College Station, TX 77843, USA.ORCID 0000-0002-8796-8123
Mihye AhnUniversity of Nevada-Reno, Reno, NV 89557, USA.ORCID 0000-0002-0388-7036
Hui MengMedical College of Wisconsin, Milwaukee, WI 53226, USA.ORCID 0000-0002-9864-4183
Margaret J BeatkaMedical College of Wisconsin, Milwaukee, WI 53226, USA.
Brittany A FickauMedical College of Wisconsin, Milwaukee, WI 53226, USA.ORCID 0000-0003-1616-6761
Juan C PrietoUniversity of North Carolina, Chapel Hill, NC 27599, USA.ORCID 0000-0002-3778-9098
Martin A StynerUniversity of North Carolina, Chapel Hill, NC 27599, USA.ORCID 0000-0002-8747-5118
Michael J StruharikSolid Biosciences Inc., Cambridge, MA 02142, USA.ORCID 0000-0001-5681-4300
Courtney ShanksSolid Biosciences Inc., Cambridge, MA 02142, USA.
Kristy J BrownSolid Biosciences Inc., Cambridge, MA 02142, USA.
Diane GolebiowskiSolid Biosciences Inc., Cambridge, MA 02142, USA.ORCID 0000-0002-5550-7883
Amanda K BettisTexas A&M University, College of Veterinary Medicine and Biomedical Sciences, College Station, TX 77843, USA.ORCID 0000-0002-2904-9268
Cynthia J Balog-AlvarezTexas A&M University, College of Veterinary Medicine and Biomedical Sciences, College Station, TX 77843, USA.ORCID 0000-0003-0426-8066
Nathalie ClementPowell Gene Therapy Center, University of Florida, Gainesville, FL 32610, USA.
Kirsten E ColemanPowell Gene Therapy Center, University of Florida, Gainesville, FL 32610, USA.ORCID 0000-0002-6289-4711
Manuela CortiPowell Gene Therapy Center, University of Florida, Gainesville, FL 32610, USA.
Xiufang PanSchool of Medicine, University of Missouri, Columbia, MO 65212, USA.ORCID 0000-0002-2594-5911
Stephen D HauschkaUniversity of Washington, Seattle, WA 98109, USA.ORCID 0000-0003-2425-2766
J Patrick GonzalezSolid Biosciences Inc., Cambridge, MA 02142, USA.
Carl A MorrisSolid Biosciences Inc., Cambridge, MA 02142, USA.ORCID 0000-0002-0973-4268
Joel S SchneiderSolid Biosciences Inc., Cambridge, MA 02142, USA.
Dongsheng DuanSchool of Medicine, University of Missouri, Columbia, MO 65212, USA.ORCID 0000-0003-4109-1132
Jeffrey S ChamberlainUniversity of Washington, Seattle, WA 98109, USA.ORCID 0000-0001-5299-0059
Barry J ByrnePowell Gene Therapy Center, University of Florida, Gainesville, FL 32610, USA.ORCID 0000-0002-7302-1756
Joe N KornegayTexas A&M University, College of Veterinary Medicine and Biomedical Sciences, College Station, TX 77843, USA.ORCID 0000-0002-5594-1882
Solidus Biosciences (United States) · USTexas A&M University · USUniversity of Florida · USMedical College of Wisconsin · USUniversity of Washington · USUniversity of Missouri · USUniversity of North Carolina at Chapel Hill · USNorth Carolina State University · USUniversity of Nevada, Reno · US
Funding
Wellstone Muscular Dystrophy Specialized Research Center (Seattle)P50AR065139 · NIAMS · UNIVERSITY OF WASHINGTON · PI JEFFREY S CHAMBERLAIN · 2018 to 2026
$15.9MDYSTROPHIN 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 P50 AR065139NIAMS NIH HHS R01 AR040864NINDS NIH HHS R01 NS090634NINDS NIH HHS R01 NS117912
Abstract
Duchenne muscular dystrophy (DMD) is a progressive muscle wasting disease caused by the absence of dystrophin, a membrane-stabilizing protein encoded by the
Indexed as
Muscular Dystrophy, AnimalMuscular Dystrophy, DuchenneAnimalsDogsDystrophinGenetic TherapyHeartHumansInfant, NewbornMiceMusclesMuscle, SkeletalDystrophin
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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.