ArticleNature medicine2020
Somatic gene editing ameliorates skeletal and cardiac muscle failure in pig and human models of Duchenne muscular dystrophy.
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Article in Nature medicine, 2020. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 166 papers, 1 of them a synthesis that pooled it.
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166citing papers in PubMed, 1 pooled it
14.4field-weighted citation impact, top 1% of its field
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Who cites it
166 citing papers in PubMed, 1 synthesis or guideline pooled it, 234 citations in OpenAlex.
- Muscle and cardiac therapeutic strategies for Duchenne muscular dystrophy: past, present, and future.Pharmacological reports : PR · 2020Pooled it
- Article
- Activating a patient-relevant pathogenic variant of a genetically defined heart disease in a humanized pig model.Lab animal · 2026Article
- Systemic regeneration medicines for muscular dystrophy: progress and challenges in pro-regenerative drug development.Cell regeneration (London, England) · 2026Review
- Advances in Living Myocardial Slice Technology for Heart Failure Research.Current heart failure reports · 2026Review
- Models of the human heart for biomedical research: Opportunities and challenges.Physiological reports · 2026Review
- Protocol for in vivo analysis of muscle function in porcine models for muscular dystrophies.STAR protocols · 2026Article
- Current Trends in Duchenne Muscular Dystrophy Research and Therapy: 3D Cardiac Modelling.Journal of cachexia, sarcopenia and muscle · 2026Review
- Review
- Genetic strategies for therapy of Duchenne muscular dystrophy.Molecular therapy. Nucleic acids · 2025Review
- Gene Editing Applications as Future Cardiovascular Therapies.Annual review of genetics · 2025Review
- Genotype and phenotype correlation analysis in 27 families with multiple osteochondroma and validation by ATDC5 chondrocyte models.Bone & joint research · 2025Article
- Cold Exposure Induces Swine Brown Adipocytes to Display an Island-like Distribution with Atypical Characteristics.International journal of molecular sciences · 2025Article
- Peeling back the layers of immunogenicity in Cas9-based genomic medicine.Molecular therapy : the journal of the American Society of Gene Therapy · 2025Review
- Insights into tissue- and cell type-specific effects of Grb10 on pig skeletal muscle growth by multi-omics analysis.Science China. Life sciences · 2025Article
- Review
- Engineering Targeted Gene Delivery Systems for Primary Hereditary Skeletal Myopathies: Current Strategies and Future Perspectives.Biomedicines · 2025Review
- Beyond the Cut: Long-read sequencing reveals complex genomic and transcriptomic changes in AAV-CRISPR therapy for Duchenne Muscular Dystrophy.bioRxiv : the preprint server for biology · 2025Article
- Pantethine ameliorates dilated cardiomyopathy features in PPCS deficiency disorder in patients and cell line models.Communications medicine · 2025Article
- Duchenne Muscular Dystrophy: Integrating Current Clinical Practice with Future Therapeutic and Diagnostic Horizons.International journal of molecular sciences · 2025Review
106 more citing papers are in PubMed but not listed here.
Corrections and comments
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Authors and funding
44 authors at 6 institutions in 1 country.
A Moretti *Klinik und Poliklinik für Innere Medizin I, Klinikum rechts der Isar, Technical University Munich and German Center for Cardiovascular Research (DZHK), Munich Heart Alliance, Munich, Germany. amoretti@mytum.de.ORCID http://orcid.org/0000-0001-5782-7832
L Fonteyne *Chair for Molecular Animal Breeding and Biotechnology, Gene Center and Department of Veterinary Sciences, LMU Munich, Munich, Germany.
F Giesert *Institute of Developmental Genetics, Helmholtz Centre and Munich School of Life Sciences Weihenstephan, Technical University of Munich, Munich, Germany.
P Hoppmann *Klinik und Poliklinik für Innere Medizin I, Klinikum rechts der Isar, Technical University Munich and German Center for Cardiovascular Research (DZHK), Munich Heart Alliance, Munich, Germany.
A B Meier *Klinik und Poliklinik für Innere Medizin I, Klinikum rechts der Isar, Technical University Munich and German Center for Cardiovascular Research (DZHK), Munich Heart Alliance, Munich, Germany.
T BozogluKlinik und Poliklinik für Innere Medizin I, Klinikum rechts der Isar, Technical University Munich and German Center for Cardiovascular Research (DZHK), Munich Heart Alliance, Munich, Germany.
A BaehrKlinik und Poliklinik für Innere Medizin I, Klinikum rechts der Isar, Technical University Munich and German Center for Cardiovascular Research (DZHK), Munich Heart Alliance, Munich, Germany.
C M SchneiderKlinik und Poliklinik für Innere Medizin I, Klinikum rechts der Isar, Technical University Munich and German Center for Cardiovascular Research (DZHK), Munich Heart Alliance, Munich, Germany.
D SinneckerKlinik und Poliklinik für Innere Medizin I, Klinikum rechts der Isar, Technical University Munich and German Center for Cardiovascular Research (DZHK), Munich Heart Alliance, Munich, Germany.ORCID http://orcid.org/0000-0002-8116-9750
K KlettKlinik und Poliklinik für Innere Medizin I, Klinikum rechts der Isar, Technical University Munich and German Center for Cardiovascular Research (DZHK), Munich Heart Alliance, Munich, Germany.
T FröhlichChair for Molecular Animal Breeding and Biotechnology, Gene Center and Department of Veterinary Sciences, LMU Munich, Munich, Germany.
F Abdel RahmanKlinik und Poliklinik für Innere Medizin I, Klinikum rechts der Isar, Technical University Munich and German Center for Cardiovascular Research (DZHK), Munich Heart Alliance, Munich, Germany.
T HaufeKlinik und Poliklinik für Innere Medizin I, Klinikum rechts der Isar, Technical University Munich and German Center for Cardiovascular Research (DZHK), Munich Heart Alliance, Munich, Germany.
S SunKlinik und Poliklinik für Innere Medizin I, Klinikum rechts der Isar, Technical University Munich and German Center for Cardiovascular Research (DZHK), Munich Heart Alliance, Munich, Germany.ORCID http://orcid.org/0000-0002-9584-9909
V JurischKlinik und Poliklinik für Innere Medizin I, Klinikum rechts der Isar, Technical University Munich and German Center for Cardiovascular Research (DZHK), Munich Heart Alliance, Munich, Germany.
B KesslerChair for Molecular Animal Breeding and Biotechnology, Gene Center and Department of Veterinary Sciences, LMU Munich, Munich, Germany.
R HinkelKlinik und Poliklinik für Innere Medizin I, Klinikum rechts der Isar, Technical University Munich and German Center for Cardiovascular Research (DZHK), Munich Heart Alliance, Munich, Germany.
R DirschingerKlinik und Poliklinik für Innere Medizin I, Klinikum rechts der Isar, Technical University Munich and German Center for Cardiovascular Research (DZHK), Munich Heart Alliance, Munich, Germany.
E MartensKlinik und Poliklinik für Innere Medizin I, Klinikum rechts der Isar, Technical University Munich and German Center for Cardiovascular Research (DZHK), Munich Heart Alliance, Munich, Germany.
C JilekKlinik und Poliklinik für Innere Medizin I, Klinikum rechts der Isar, Technical University Munich and German Center for Cardiovascular Research (DZHK), Munich Heart Alliance, Munich, Germany.
A GrafChair for Molecular Animal Breeding and Biotechnology, Gene Center and Department of Veterinary Sciences, LMU Munich, Munich, Germany.ORCID http://orcid.org/0000-0003-4435-6861
S KrebsChair for Molecular Animal Breeding and Biotechnology, Gene Center and Department of Veterinary Sciences, LMU Munich, Munich, Germany.
G SantamariaKlinik und Poliklinik für Innere Medizin I, Klinikum rechts der Isar, Technical University Munich and German Center for Cardiovascular Research (DZHK), Munich Heart Alliance, Munich, Germany.
M KuromeChair for Molecular Animal Breeding and Biotechnology, Gene Center and Department of Veterinary Sciences, LMU Munich, Munich, Germany.
V ZakhartchenkoChair for Molecular Animal Breeding and Biotechnology, Gene Center and Department of Veterinary Sciences, LMU Munich, Munich, Germany.
B CampbellKlinik und Poliklinik für Innere Medizin I, Klinikum rechts der Isar, Technical University Munich and German Center for Cardiovascular Research (DZHK), Munich Heart Alliance, Munich, Germany.
K VoelseReseach Unit Apoptosis in Hemopoietic Stem Cells, Helmholtz Zentrum München, German Center for Environmental Health (HMGU), Munich, Germany.
A WolfKlinik und Poliklinik für Innere Medizin I, Klinikum rechts der Isar, Technical University Munich and German Center for Cardiovascular Research (DZHK), Munich Heart Alliance, Munich, Germany.
T ZieglerKlinik und Poliklinik für Innere Medizin I, Klinikum rechts der Isar, Technical University Munich and German Center for Cardiovascular Research (DZHK), Munich Heart Alliance, Munich, Germany.
S ReichertDepartment of Neurology, Friedrich Baur Institute, LMU Munich, Munich, Germany.
S LeeKlinik und Poliklinik für Innere Medizin I, Klinikum rechts der Isar, Technical University Munich and German Center for Cardiovascular Research (DZHK), Munich Heart Alliance, Munich, Germany.
F FlenkenthalerChair for Molecular Animal Breeding and Biotechnology, Gene Center and Department of Veterinary Sciences, LMU Munich, Munich, Germany.
T DornKlinik und Poliklinik für Innere Medizin I, Klinikum rechts der Isar, Technical University Munich and German Center for Cardiovascular Research (DZHK), Munich Heart Alliance, Munich, Germany.
I JeremiasReseach Unit Apoptosis in Hemopoietic Stem Cells, Helmholtz Zentrum München, German Center for Environmental Health (HMGU), Munich, Germany.ORCID http://orcid.org/0000-0003-1773-7677
H BlumChair for Molecular Animal Breeding and Biotechnology, Gene Center and Department of Veterinary Sciences, LMU Munich, Munich, Germany.
A DendorferWalter Brendel Centre of Experimental Medicine, University Hospital, LMU Munich, Munich, Germany.
A SchniekeChair of Livestock Biotechnology, School of Life Sciences Weihenstephan, Technical University of Munich, Munich, Germany.
S KrauseDepartment of Neurology, Friedrich Baur Institute, LMU Munich, Munich, Germany.
M C WalterDepartment of Neurology, Friedrich Baur Institute, LMU Munich, Munich, Germany.
N KlymiukChair for Molecular Animal Breeding and Biotechnology, Gene Center and Department of Veterinary Sciences, LMU Munich, Munich, Germany.
K L LaugwitzKlinik und Poliklinik für Innere Medizin I, Klinikum rechts der Isar, Technical University Munich and German Center for Cardiovascular Research (DZHK), Munich Heart Alliance, Munich, Germany.
E WolfChair for Molecular Animal Breeding and Biotechnology, Gene Center and Department of Veterinary Sciences, LMU Munich, Munich, Germany.
W WurstInstitute of Developmental Genetics, Helmholtz Centre and Munich School of Life Sciences Weihenstephan, Technical University of Munich, Munich, Germany.
C KupattKlinik und Poliklinik für Innere Medizin I, Klinikum rechts der Isar, Technical University Munich and German Center for Cardiovascular Research (DZHK), Munich Heart Alliance, Munich, Germany. christian.kupatt@tum.de.ORCID http://orcid.org/0000-0002-3611-1218
TUM Klinikum · DELudwig-Maximilians-Universität München · DEFriedrich Baur Stiftung · DEHelmholtz Zentrum München · DETechnical University of Munich · DEGerman Center for Neurodegenerative Diseases · DE
Funding
European Research Council 681524European Research Council 788381
Abstract
Frameshift mutations in the DMD gene, encoding dystrophin, cause Duchenne muscular dystrophy (DMD), leading to terminal muscle and heart failure in patients. Somatic gene editing by sequence-specific nucleases offers new options for restoring the DMD reading frame, resulting in expression of a shortened but largely functional dystrophin protein. Here, we validated this approach in a pig model of DMD lacking exon 52 of DMD (DMDΔ52), as well as in a corresponding patient-derived induced pluripotent stem cell model. In DMDΔ52 pigs
Indexed as
Frameshift MutationAnimalsDisease Models, AnimalDystrophinExonsFemaleGene EditingGene Expression RegulationGenetic TherapyGenomeHeart FailureHumansInduced Pluripotent Stem CellsMaleMass SpectrometryMusclesDystrophinProteomeRNA, Guide, CRISPR-Cas Systems
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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.