ArticleScience (New York, N.Y.)2018
Gene editing restores dystrophin expression in a canine model of Duchenne muscular dystrophy.
Open access · greenAbstract read
In one paragraph
Article in Science (New York, N.Y.), 2018. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 329 papers, 1 of them a synthesis that pooled it.
0numbers the graph read from it
0cells of the map it votes in
329citing papers in PubMed, 1 pooled it
27.6field-weighted citation impact, top 1% of its field
What it found
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Who cites it
329 citing papers in PubMed, 1 synthesis or guideline pooled it, 541 citations in OpenAlex.
- Dilated cardiomyopathy as the initial presentation of Becker muscular dystrophy: a systematic review of published cases.Orphanet journal of rare diseases · 2022Pooled it
- Patient-derived teratomas as a humanized platform for dystrophin restoration by ex vivo cell therapy and in vivo base editing.Experimental & molecular medicine · 2026Article
- CRISPR-Cas9-mediated upregulation of utrophin ameliorates Duchenne muscular dystrophy.Molecular therapy : the journal of the American Society of Gene Therapy · 2026Article
- SORT LNPs encapsulating Cas9 mRNA achieve efficient editing in skeletal muscle in a dystrophic mouse model.Molecular therapy : the journal of the American Society of Gene Therapy · 2026Article
- Muscle-targeted extracellular vesicles for full-length dystrophin mRNA therapy in Duchenne muscular dystrophy.Nature biomedical engineering · 2026Article
- A primer on prime: A prime editing update from advances to first-in-human trial.Molecular therapy : the journal of the American Society of Gene Therapy · 2026Review
- Gene Editing Strategies for Duchenne Muscular Dystrophy: From Molecular Mechanisms to Clinical Translation.Cells · 2026Review
- CRISPR-mediated targeting of theMolecular therapy. Advances · 2026Article
- Intravenous rAAV9 Produces Time-Resolved Parenchymal Labeling Downstream of the Vasculature in Adult Mice.Biology · 2026Article
- Advances in Double-Stranded DNA Targeting Technologies.Exploration (Beijing, China) · 2026Review
- Developing therapeutics for rare cardiovascular diseases.American heart journal · 2026Review
- Canine Model of Duchenne Muscular Dystrophy.Methods in molecular biology (Clifton, N.J.) · 2026Article
- Targeted delivery of genome editors in vivo.Nature biotechnology · 2026Review
- Genetic strategies for therapy of Duchenne muscular dystrophy.Molecular therapy. Nucleic acids · 2025Review
- Megabase-scale loss of heterozygosity provoked by CRISPR-Cas9 DNA double-strand breaks.Molecular cell · 2025Article
- Gene Editing Applications as Future Cardiovascular Therapies.Annual review of genetics · 2025Review
- Emerging lipid nanoparticle systems capable of efficient intramuscular RNA delivery.Nanomedicine (London, England) · 2025Review
- Peeling back the layers of immunogenicity in Cas9-based genomic medicine.Molecular therapy : the journal of the American Society of Gene Therapy · 2025Review
- Engineering adeno-associated viral vectors for CRISPR/Cas based in vivo therapeutic genome editing.Biomaterials · 2025Review
- Stable Cas9 expression regulates cell growth by facilitating mTORC2 activation.Nucleic acids research · 2025Article
269 more citing papers are in PubMed but not listed here.
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Authors and funding
13 authors at 3 institutions in 2 countries.
Leonela AmoasiiDepartment of Molecular Biology, Hamon Center for Regenerative Science and Medicine, Sen. Paul D. Wellstone Muscular Dystrophy Cooperative Research Center, University of Texas Southwestern Medical Center, 5323 Harry Hines Boulevard, Dallas, TX 75390, USA.ORCID 0000-0001-7960-2749
John C W HildyardDepartment of Clinical Science and Services, Comparative Neuromuscular Diseases Laboratory, Royal Veterinary College, London NW1 0TU, UK.ORCID 0000-0003-2283-2118
Hui LiDepartment of Molecular Biology, Hamon Center for Regenerative Science and Medicine, Sen. Paul D. Wellstone Muscular Dystrophy Cooperative Research Center, University of Texas Southwestern Medical Center, 5323 Harry Hines Boulevard, Dallas, TX 75390, USA.ORCID 0000-0002-4114-8349
Efrain Sanchez-OrtizDepartment of Molecular Biology, Hamon Center for Regenerative Science and Medicine, Sen. Paul D. Wellstone Muscular Dystrophy Cooperative Research Center, University of Texas Southwestern Medical Center, 5323 Harry Hines Boulevard, Dallas, TX 75390, USA.ORCID 0000-0001-9358-1630
Alex MireaultDepartment of Molecular Biology, Hamon Center for Regenerative Science and Medicine, Sen. Paul D. Wellstone Muscular Dystrophy Cooperative Research Center, University of Texas Southwestern Medical Center, 5323 Harry Hines Boulevard, Dallas, TX 75390, USA.ORCID 0000-0002-7534-636X
Daniel CaballeroDepartment of Molecular Biology, Hamon Center for Regenerative Science and Medicine, Sen. Paul D. Wellstone Muscular Dystrophy Cooperative Research Center, University of Texas Southwestern Medical Center, 5323 Harry Hines Boulevard, Dallas, TX 75390, USA.ORCID 0000-0001-9232-8991
Rachel HarronDepartment of Clinical Science and Services, Comparative Neuromuscular Diseases Laboratory, Royal Veterinary College, London NW1 0TU, UK.ORCID 0000-0001-9418-0708
Thaleia-Rengina StathopoulouSection of Anaesthesia and Analgesia, Royal Veterinary College, London NW1 0TU, UK.ORCID 0000-0003-4666-2002
Claire MasseyDepartment of Clinical Science and Services, Comparative Neuromuscular Diseases Laboratory, Royal Veterinary College, London NW1 0TU, UK.ORCID 0000-0003-0823-6547
John M SheltonDepartment of Internal Medicine, University of Texas Southwestern Medical Center, 5323 Harry Hines Boulevard, Dallas, TX 75390, USA.ORCID 0000-0002-2949-1638
Rhonda Bassel-DubyDepartment of Molecular Biology, Hamon Center for Regenerative Science and Medicine, Sen. Paul D. Wellstone Muscular Dystrophy Cooperative Research Center, University of Texas Southwestern Medical Center, 5323 Harry Hines Boulevard, Dallas, TX 75390, USA.ORCID 0000-0001-7568-410X
Richard J PiercyDepartment of Clinical Science and Services, Comparative Neuromuscular Diseases Laboratory, Royal Veterinary College, London NW1 0TU, UK.ORCID 0000-0002-4344-6438
Eric N OlsonDepartment of Molecular Biology, Hamon Center for Regenerative Science and Medicine, Sen. Paul D. Wellstone Muscular Dystrophy Cooperative Research Center, University of Texas Southwestern Medical Center, 5323 Harry Hines Boulevard, Dallas, TX 75390, USA. eric.olson@utsouthwestern.edu.ORCID 0000-0003-1151-8262
The University of Texas Southwestern Medical Center · USRoyal Veterinary College · GBCentrexion Therapeutics (United States) · US
Funding
Training CoreU54HD087351 · NICHD · UT SOUTHWESTERN MEDICAL CENTER · PI OLSON, ERIC N · 2015 to 2019
$7.8MDeciphering the role of a novel micropeptide in cardiac function and dysfunctionR01HL130253 · NHLBI · UT SOUTHWESTERN MEDICAL CENTER · PI BASSEL-DUBY, RHONDA, OLSON, ERIC N · 2016 to 2023
$4.4MMolecular Dissection of Myoblast Fusion In Muscle Development and RegenerationR01AR067294 · NIAMS · UT SOUTHWESTERN MEDICAL CENTER · PI BASSEL-DUBY, RHONDA, OLSON, ERIC N · 2015 to 2019
$1.8MNHLBI NIH HHS R01 HL130253NIAMS NIH HHS R01 AR067294NICHD NIH HHS U54 HD087351Wellcome Trust
Abstract
Mutations in the gene encoding dystrophin, a protein that maintains muscle integrity and function, cause Duchenne muscular dystrophy (DMD). The deltaE50-MD dog model of DMD harbors a mutation corresponding to a mutational "hotspot" in the human
Indexed as
AdenoviridaeAnimalsCRISPR-Cas SystemsDisease Models, AnimalDogsDystrophinFemaleGene EditingGene Transfer TechniquesMaleMuscular Dystrophy, DuchenneDystrophin
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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.