{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,9,16]],"date-time":"2026-09-16T12:59:22Z","timestamp":1789563562655,"version":"build-2803163510"},"reference-count":94,"publisher":"MDPI AG","issue":"23","license":[{"start":{"date-parts":[[2024,12,6]],"date-time":"2024-12-06T00:00:00Z","timestamp":1733443200000},"content-version":"vor","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by\/4.0\/"}],"content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":["IJMS"],"abstract":"<jats:p>For years, the treatment of many cardiomyopathies has been solely focused on symptom management. However, cardiomyopathies have a genetic substrate, and directing therapy towards the pathophysiology rather than the epiphenomenon of the disease may be a winning strategy. Gene therapy involves the insertion of genes or the modification of existing ones and their regulatory elements through strategies like gene replacement and gene editing. Recently, gene therapy for cardiac amyloidosis and Duchenne muscular dystrophy has received approval, and important clinical trials are currently evaluating gene therapy methods for rare heart diseases like Friedreich\u2019s Ataxia, Danon disease, Fabry disease, and Pompe Disease. Furthermore, favorable results have been noted in animal studies receiving gene therapy for hypertrophic, dilated, and arrhythmogenic cardiomyopathy. This review discusses gene therapy methods, ongoing clinical trials, and future goals in this area.<\/jats:p>","DOI":"10.3390\/ijms252313147","type":"journal-article","created":{"date-parts":[[2024,12,6]],"date-time":"2024-12-06T10:58:45Z","timestamp":1733482725000},"page":"13147","update-policy":"https:\/\/doi.org\/10.3390\/mdpi_crossmark_policy","source":"Crossref","is-referenced-by-count":9,"title":["Unveiling the Future of Cardiac Care: A Review of Gene Therapy in Cardiomyopathies"],"prefix":"10.3390","volume":"25","author":[{"ORCID":"https:\/\/orcid.org\/0009-0000-0874-9735","authenticated-orcid":false,"given":"Damiano","family":"Venturiello","sequence":"first","affiliation":[{"name":"Cardiology, Clinical and Molecular Medicine Department, Sapienza University of Rome, 00189 Rome, Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Pier Giorgio","family":"Tiberi","sequence":"additional","affiliation":[{"name":"Cardiology, Clinical and Molecular Medicine Department, Sapienza University of Rome, 00189 Rome, Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Francesco","family":"Perulli","sequence":"additional","affiliation":[{"name":"Cardiology, Clinical and Molecular Medicine Department, Sapienza University of Rome, 00189 Rome, Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-6861-6870","authenticated-orcid":false,"given":"Giulia","family":"Nardoianni","sequence":"additional","affiliation":[{"name":"Cardiology, Clinical and Molecular Medicine Department, Sapienza University of Rome, 00189 Rome, Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Leonardo","family":"Guida","sequence":"additional","affiliation":[{"name":"Cardiology, Clinical and Molecular Medicine Department, Sapienza University of Rome, 00189 Rome, Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0009-0001-5216-212X","authenticated-orcid":false,"given":"Carlo","family":"Barsali","sequence":"additional","affiliation":[{"name":"Cardiology, Clinical and Molecular Medicine Department, Sapienza University of Rome, 00189 Rome, Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Carlo","family":"Terrone","sequence":"additional","affiliation":[{"name":"Cardiology, Clinical and Molecular Medicine Department, Sapienza University of Rome, 00189 Rome, Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Alessandro","family":"Cianca","sequence":"additional","affiliation":[{"name":"Cardiology, Clinical and Molecular Medicine Department, Sapienza University of Rome, 00189 Rome, Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Camilla","family":"Lustri","sequence":"additional","affiliation":[{"name":"Cardiology, Clinical and Molecular Medicine Department, Sapienza University of Rome, 00189 Rome, Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Matteo","family":"Sclafani","sequence":"additional","affiliation":[{"name":"Cardiology, Clinical and Molecular Medicine Department, Sapienza University of Rome, 00189 Rome, Italy"},{"name":"Royal Brompton and Harefield Hospitals, Guy\u2019s and St Thomas\u2019 NHS Foundation Trust, London SW3 6PY, UK"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-8520-152X","authenticated-orcid":false,"given":"Giacomo","family":"Tini","sequence":"additional","affiliation":[{"name":"Cardiology, Clinical and Molecular Medicine Department, Sapienza University of Rome, 00189 Rome, Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Emanuele","family":"Barbato","sequence":"additional","affiliation":[{"name":"Cardiology, Clinical and Molecular Medicine Department, Sapienza University of Rome, 00189 Rome, Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-4872-7981","authenticated-orcid":false,"given":"Beatrice","family":"Musumeci","sequence":"additional","affiliation":[{"name":"Cardiology, Clinical and Molecular Medicine Department, Sapienza University of Rome, 00189 Rome, Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"1968","published-online":{"date-parts":[[2024,12,6]]},"reference":[{"key":"ref_1","first-page":"778","article-title":"Cardiomyopathy: An Overview","volume":"79","author":"Wexler","year":"2010","journal-title":"Am. 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