ArticleCirculation research2017
In Vivo Genome Editing Restores Dystrophin Expression and Cardiac Function in Dystrophic Mice.
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Article in Circulation research, 2017. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 88 papers, 2 of them syntheses that pooled it.
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88citing papers in PubMed, 2 pooled it
7.6field-weighted citation impact, top 2% of its field
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Who cites it
88 citing papers in PubMed, 2 syntheses or guidelines pooled it, 142 citations in OpenAlex.
- Muscle and cardiac therapeutic strategies for Duchenne muscular dystrophy: past, present, and future.Pharmacological reports : PR · 2020Pooled it
- CRISPR-cas gene-editing as plausible treatment of neuromuscular and nucleotide-repeat-expansion diseases: A systematic review.PloS one · 2019Pooled it
- A Translational Roadmap for Neurological Nonsense Mutation Disorders.International journal of molecular sciences · 2026Review
- Genetic strategies for therapy of Duchenne muscular dystrophy.Molecular therapy. Nucleic acids · 2025Review
- CRISPR Technology in Disease Management: An Updated Review of Clinical Translation and Therapeutic Potential.Cell proliferation · 2025Article
- Preclinical development of genome editing to treat Duchenne muscular dystrophy by exon skipping.Journal of neuromuscular diseases · 2025Review
- Treating neuromuscular diseases: unveiling gene therapy breakthroughs and pioneering future applications.Journal of biomedical science · 2025Review
- AFrontiers in genetics · 2025Article
- Review
- Circadian Clock in Muscle Disease Etiology and Therapeutic Potential for Duchenne Muscular Dystrophy.International journal of molecular sciences · 2024Review
- Cardiomyopathies in children and adolescents: aetiology, management, and outcomes in the European Society of Cardiology EURObservational Research Programme Cardiomyopathy and Myocarditis Registry.European heart journal · 2024Article
- Breaking genetic shackles: The advance of base editing in genetic disorder treatment.Frontiers in pharmacology · 2024Review
- Dystrophin- and Utrophin-Based Therapeutic Approaches for Treatment of Duchenne Muscular Dystrophy: A Comparative Review.BioDrugs : clinical immunotherapeutics, biopharmaceuticals and gene therapy · 2024Review
- What did CRISPR-Cas9 accomplish in its first 10 years?Biochemia medica · 2023Article
- Dual CRISPR-Cas3 system for inducing multi-exon skipping in DMD patient-derived iPSCs.Stem cell reports · 2023Article
- CRISPR-Cas Technology as a Revolutionary Genome Editing tool: Mechanisms and Biomedical Applications.Iranian biomedical journal · 2023Review
- Systemic deletion ofProceedings of the National Academy of Sciences of the United States of America · 2023Article
- Liver-specific in vivo base editing of Angptl3 via AAV delivery efficiently lowers blood lipid levels in mice.Cell & bioscience · 2023Article
- Article
- Gene Modulation with CRISPR-based Tools in Human iPSC-Cardiomyocytes.Stem cell reviews and reports · 2023Review
28 more citing papers are in PubMed but not listed here.
Corrections and comments
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Authors and funding
12 authors at 1 institution in 1 country.
Mona El RefaeyFrom the Department of Surgery, Davis Heart and Lung Research Institute, Biomedical Sciences Graduate Program, Biophysics Graduate Program (M.E.R., L.X., Y.G., T.M.A.A., J.M., R.H.) and Department of Physiology and Cell Biology, Davis Heart and Lung Research Institute (B.D.C., P.J.M., P.M.L.J.), The Ohio State University Wexner Medical Center, Columbus; Genomics Shared Resource, The Ohio State University Comprehensive Cancer Center, Columbus (S.C.W., D.E.S.); and Department of Cancer Biology and Genetics (K.A., D.E.S.) and Human Cancer Genetics Program, and Department of Biomedical Informatics (adjunct) (D.E.S.), The Ohio State University, Columbus.
Li XuFrom the Department of Surgery, Davis Heart and Lung Research Institute, Biomedical Sciences Graduate Program, Biophysics Graduate Program (M.E.R., L.X., Y.G., T.M.A.A., J.M., R.H.) and Department of Physiology and Cell Biology, Davis Heart and Lung Research Institute (B.D.C., P.J.M., P.M.L.J.), The Ohio State University Wexner Medical Center, Columbus; Genomics Shared Resource, The Ohio State University Comprehensive Cancer Center, Columbus (S.C.W., D.E.S.); and Department of Cancer Biology and Genetics (K.A., D.E.S.) and Human Cancer Genetics Program, and Department of Biomedical Informatics (adjunct) (D.E.S.), The Ohio State University, Columbus.
Yandi GaoFrom the Department of Surgery, Davis Heart and Lung Research Institute, Biomedical Sciences Graduate Program, Biophysics Graduate Program (M.E.R., L.X., Y.G., T.M.A.A., J.M., R.H.) and Department of Physiology and Cell Biology, Davis Heart and Lung Research Institute (B.D.C., P.J.M., P.M.L.J.), The Ohio State University Wexner Medical Center, Columbus; Genomics Shared Resource, The Ohio State University Comprehensive Cancer Center, Columbus (S.C.W., D.E.S.); and Department of Cancer Biology and Genetics (K.A., D.E.S.) and Human Cancer Genetics Program, and Department of Biomedical Informatics (adjunct) (D.E.S.), The Ohio State University, Columbus.
Benjamin D CananFrom the Department of Surgery, Davis Heart and Lung Research Institute, Biomedical Sciences Graduate Program, Biophysics Graduate Program (M.E.R., L.X., Y.G., T.M.A.A., J.M., R.H.) and Department of Physiology and Cell Biology, Davis Heart and Lung Research Institute (B.D.C., P.J.M., P.M.L.J.), The Ohio State University Wexner Medical Center, Columbus; Genomics Shared Resource, The Ohio State University Comprehensive Cancer Center, Columbus (S.C.W., D.E.S.); and Department of Cancer Biology and Genetics (K.A., D.E.S.) and Human Cancer Genetics Program, and Department of Biomedical Informatics (adjunct) (D.E.S.), The Ohio State University, Columbus.
T M Ayodele AdesanyaFrom the Department of Surgery, Davis Heart and Lung Research Institute, Biomedical Sciences Graduate Program, Biophysics Graduate Program (M.E.R., L.X., Y.G., T.M.A.A., J.M., R.H.) and Department of Physiology and Cell Biology, Davis Heart and Lung Research Institute (B.D.C., P.J.M., P.M.L.J.), The Ohio State University Wexner Medical Center, Columbus; Genomics Shared Resource, The Ohio State University Comprehensive Cancer Center, Columbus (S.C.W., D.E.S.); and Department of Cancer Biology and Genetics (K.A., D.E.S.) and Human Cancer Genetics Program, and Department of Biomedical Informatics (adjunct) (D.E.S.), The Ohio State University, Columbus.
Sarah C WarnerFrom the Department of Surgery, Davis Heart and Lung Research Institute, Biomedical Sciences Graduate Program, Biophysics Graduate Program (M.E.R., L.X., Y.G., T.M.A.A., J.M., R.H.) and Department of Physiology and Cell Biology, Davis Heart and Lung Research Institute (B.D.C., P.J.M., P.M.L.J.), The Ohio State University Wexner Medical Center, Columbus; Genomics Shared Resource, The Ohio State University Comprehensive Cancer Center, Columbus (S.C.W., D.E.S.); and Department of Cancer Biology and Genetics (K.A., D.E.S.) and Human Cancer Genetics Program, and Department of Biomedical Informatics (adjunct) (D.E.S.), The Ohio State University, Columbus.
Keiko AkagiFrom the Department of Surgery, Davis Heart and Lung Research Institute, Biomedical Sciences Graduate Program, Biophysics Graduate Program (M.E.R., L.X., Y.G., T.M.A.A., J.M., R.H.) and Department of Physiology and Cell Biology, Davis Heart and Lung Research Institute (B.D.C., P.J.M., P.M.L.J.), The Ohio State University Wexner Medical Center, Columbus; Genomics Shared Resource, The Ohio State University Comprehensive Cancer Center, Columbus (S.C.W., D.E.S.); and Department of Cancer Biology and Genetics (K.A., D.E.S.) and Human Cancer Genetics Program, and Department of Biomedical Informatics (adjunct) (D.E.S.), The Ohio State University, Columbus.
David E SymerFrom the Department of Surgery, Davis Heart and Lung Research Institute, Biomedical Sciences Graduate Program, Biophysics Graduate Program (M.E.R., L.X., Y.G., T.M.A.A., J.M., R.H.) and Department of Physiology and Cell Biology, Davis Heart and Lung Research Institute (B.D.C., P.J.M., P.M.L.J.), The Ohio State University Wexner Medical Center, Columbus; Genomics Shared Resource, The Ohio State University Comprehensive Cancer Center, Columbus (S.C.W., D.E.S.); and Department of Cancer Biology and Genetics (K.A., D.E.S.) and Human Cancer Genetics Program, and Department of Biomedical Informatics (adjunct) (D.E.S.), The Ohio State University, Columbus.
Peter J MohlerFrom the Department of Surgery, Davis Heart and Lung Research Institute, Biomedical Sciences Graduate Program, Biophysics Graduate Program (M.E.R., L.X., Y.G., T.M.A.A., J.M., R.H.) and Department of Physiology and Cell Biology, Davis Heart and Lung Research Institute (B.D.C., P.J.M., P.M.L.J.), The Ohio State University Wexner Medical Center, Columbus; Genomics Shared Resource, The Ohio State University Comprehensive Cancer Center, Columbus (S.C.W., D.E.S.); and Department of Cancer Biology and Genetics (K.A., D.E.S.) and Human Cancer Genetics Program, and Department of Biomedical Informatics (adjunct) (D.E.S.), The Ohio State University, Columbus.
Jianjie MaFrom the Department of Surgery, Davis Heart and Lung Research Institute, Biomedical Sciences Graduate Program, Biophysics Graduate Program (M.E.R., L.X., Y.G., T.M.A.A., J.M., R.H.) and Department of Physiology and Cell Biology, Davis Heart and Lung Research Institute (B.D.C., P.J.M., P.M.L.J.), The Ohio State University Wexner Medical Center, Columbus; Genomics Shared Resource, The Ohio State University Comprehensive Cancer Center, Columbus (S.C.W., D.E.S.); and Department of Cancer Biology and Genetics (K.A., D.E.S.) and Human Cancer Genetics Program, and Department of Biomedical Informatics (adjunct) (D.E.S.), The Ohio State University, Columbus.
Paul M L JanssenFrom the Department of Surgery, Davis Heart and Lung Research Institute, Biomedical Sciences Graduate Program, Biophysics Graduate Program (M.E.R., L.X., Y.G., T.M.A.A., J.M., R.H.) and Department of Physiology and Cell Biology, Davis Heart and Lung Research Institute (B.D.C., P.J.M., P.M.L.J.), The Ohio State University Wexner Medical Center, Columbus; Genomics Shared Resource, The Ohio State University Comprehensive Cancer Center, Columbus (S.C.W., D.E.S.); and Department of Cancer Biology and Genetics (K.A., D.E.S.) and Human Cancer Genetics Program, and Department of Biomedical Informatics (adjunct) (D.E.S.), The Ohio State University, Columbus.
Renzhi HanFrom the Department of Surgery, Davis Heart and Lung Research Institute, Biomedical Sciences Graduate Program, Biophysics Graduate Program (M.E.R., L.X., Y.G., T.M.A.A., J.M., R.H.) and Department of Physiology and Cell Biology, Davis Heart and Lung Research Institute (B.D.C., P.J.M., P.M.L.J.), The Ohio State University Wexner Medical Center, Columbus; Genomics Shared Resource, The Ohio State University Comprehensive Cancer Center, Columbus (S.C.W., D.E.S.); and Department of Cancer Biology and Genetics (K.A., D.E.S.) and Human Cancer Genetics Program, and Department of Biomedical Informatics (adjunct) (D.E.S.), The Ohio State University, Columbus. renzhi.han@osumc.edu.
The Ohio State University · US
Funding
Translational Therapeutics Research Program (TT)P30CA016058 · NCI · OHIO STATE UNIVERSITY · PI Tasleem J Padamsee · 1985 to 2026
$132.3MDefining novel mechanisms for human arrhythmiaR35HL135754 · NHLBI · OHIO STATE UNIVERSITY · PI MOHLER, PETER J. · 2017 to 2023
$6.0MMolecular and cellular functions of Ano5 in heartR01HL116546 · NHLBI · OHIO STATE UNIVERSITY · PI HAN, RENZHI · 2013 to 2022
$4.4MTraining in Congenital and Acquired Heart DiseaseT32HL098039 · NHLBI · RESEARCH INST NATIONWIDE CHILDREN'S HOSP · PI GARG, VIDU, MOHLER, PETER J. · 2009 to 2019
$2.5MMechanisms of Muscle Inflammation in Muscular DystrophyR01AR064241 · NIAMS · OHIO STATE UNIVERSITY · PI HAN, RENZHI · 2013 to 2017
$1.5MDr. Keiko Akagi, A Bioinformatics Specialist in Cancer Genomics Research at OSU Comprehensive Cancer CenterR50CA211533 · NCI · UNIVERSITY OF TX MD ANDERSON CAN CTR · PI AKAGI, KEIKO · 2016 to 2020
$608kNCI NIH HHS P30 CA016058NCI NIH HHS R50 CA211533NHLBI NIH HHS R01 HL116546NHLBI NIH HHS R35 HL135754NHLBI NIH HHS T32 HL098039NIAMS NIH HHS R01 AR064241
Abstract
rationaleDuchenne muscular dystrophy is a severe inherited form of muscular dystrophy caused by mutations in the reading frame of the dystrophin gene disrupting its protein expression. Dystrophic cardiomyopathy is a leading cause of death in Duchenne muscular dystrophy patients, and currently no effective treatment exists to halt its progression. Recent advancement in genome editing technologies offers a promising therapeutic approach in restoring dystrophin protein expression. However, the impact of this approach on Duchenne muscular dystrophy cardiac function has yet to be evaluated. Therefore, we assessed the therapeutic efficacy of CRISPR (clustered regularly interspaced short palindromic repeats)-mediated genome editing on dystrophin expression and cardiac function in
objectiveTo examine the efficiency and physiological impact of CRISPR-mediated genome editing on cardiac dystrophin expression and function in dystrophic mice. METHODS AND
resultsHere, we packaged SaCas9 (clustered regularly interspaced short palindromic repeat-associated 9 from
conclusionsThis study provides evidence for using CRISPR-based genome editing as a potential therapeutic approach for restoring dystrophic cardiomyopathy structurally and functionally.
Indexed as
Clustered Regularly Interspaced Short Palindromic RepeatsCRISPR-Cas SystemsMyocardial ContractionAnimalsCardiomyopathiesCRISPR-Associated ProteinsDependovirusDisease Models, AnimalDystrophinExonsFibrosisGene EditingGene Expression RegulationGenetic Predisposition to DiseaseGenetic TherapyGenetic Vectorsapo-dystrophin 1CRISPR-Associated ProteinsDystrophinRNA, Guide, CRISPR-Cas SystemsUtrophincardiomyopathiesdystrophingene editingmicemuscular dystrophy
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