ReviewPflugers Archiv : European journal of physiology2019
Gene therapy strategies in the treatment of hypertrophic cardiomyopathy.
Review in Pflugers Archiv : European journal of physiology, 2019. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 33 papers.
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
33 citing papers in PubMed, 69 citations in OpenAlex.
- Proteomic Analysis of Valsartan for Attenuating Disease Evolution in Early Sarcomeric Hypertrophic Cardiomyopathy (VANISH) Clinical Trial.Circulation. Heart failure · 2025Trial
- Therapeutic Ultrasound in Cardiovascular Medicine: Applications of Low-intensity Pulsed Ultrasound and Ultrasound-targeted Microbubble Destruction.Cardiovascular drugs and therapy · 2026Review
- Heart Failure in the Molecular Era: Redefining Our Understanding of Disease Mechanisms and Perspectives.Biomedicines · 2026Review
- Integrative Approaches in the Management of Hypertrophic Cardiomyopathy: A Comprehensive Review of Current Therapeutic Modalities.Biomedicines · 2025Review
- AAV9-mediated MYBPC3 gene therapy with optimized expression cassette enhances cardiac function and survival in MYBPC3 cardiomyopathy models.Nature communications · 2025Article
- Review
- Genetic Mutations and Mitochondrial Redox Signaling as Modulating Factors in Hypertrophic Cardiomyopathy: A Scoping Review.International journal of molecular sciences · 2024Article
- Current perspectives of sudden cardiac death management in hypertrophic cardiomyopathy.Heart failure reviews · 2024Review
- An overview of the treatments for hypertrophic cardiomyopathy.Frontiers in cardiovascular medicine · 2024Review
- Exploring Hypertrophic Cardiomyopathy Biomarkers through Integrated Bioinformatics Analysis: Uncovering Novel Diagnostic Candidates.Cardiology research and practice · 2024Article
- An Unbiased Screen Identified the Hsp70-BAG3 Complex as a Regulator of Myosin-Binding Protein C3.JACC. Basic to translational science · 2023Article
- Hypertrophic Cardiomyopathy: Genetic Foundations, Outcomes, Interconnections, and Their Modifiers.Medicina (Kaunas, Lithuania) · 2023Review
- Gene Therapy of Extracellular Vesicles in Cardiovascular and Metabolic Diseases.Advances in experimental medicine and biology · 2023Article
- A Narrative Review of Emerging Therapies for Hypertrophic Obstructive Cardiomyopathy.Current cardiology reviews · 2023Review
- Contemporary Therapies and Future Directions in the Management of Hypertrophic Cardiomyopathy.Cardiology and therapy · 2022Review
- Genetic variants in Colombian patients with inherited cardiac conditions.Molecular genetics & genomic medicine · 2022Article
- Review of Contemporary Invasive Treatment Approaches and Critical Appraisal of Guidelines on Hypertrophic Obstructive Cardiomyopathy: State-of-the-Art Review.Journal of clinical medicine · 2022Review
- Clinical Features and Natural History of Preadolescent Nonsyndromic Hypertrophic Cardiomyopathy.Journal of the American College of Cardiology · 2022Article
- Genetic Testing in Patients with Hypertrophic Cardiomyopathy.International journal of molecular sciences · 2021Review
- Phenotypes of hypertrophic cardiomyopathy: genetics, clinics, and modular imaging.Heart failure reviews · 2021Review
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
3 authors at 1 institution in 1 country.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Hypertrophic cardiomyopathy (HCM) is an inherited myocardial disease with an estimated prevalence of 1:200 caused by mutations in sarcomeric proteins. It is associated with hypertrophy of the left ventricle, increased interstitial fibrosis, and diastolic dysfunction for heterozygous mutation carriers. Carriers of double heterozygous, compound heterozygous, and homozygous mutations often display more severe forms of cardiomyopathies, ultimately leading to premature death. So far, there is no curative treatment against HCM, as current therapies are focused on symptoms relief by pharmacological intervention and not on the cause of HCM. In the last decade, several strategies have been developed to remove genetic defects, including genome editing, exon skipping, allele-specific silencing, spliceosome-mediated RNA trans-splicing, and gene replacement. Most of these technologies have already been tested for efficacy and efficiency in animal- or human-induced pluripotent stem cell models of HCM with promising results. We will summarize recent technological advances and their implication as gene therapy options in HCM with a special focus on treating MYBPC3 mutations and its potential for being a successful bench to bedside example.
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Registered trials
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.