{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,1]],"date-time":"2026-10-01T06:31:06Z","timestamp":1790836266651,"version":"4.1.0"},"reference-count":143,"publisher":"Oxford University Press (OUP)","issue":"9","license":[{"start":{"date-parts":[[2020,4,22]],"date-time":"2020-04-22T00:00:00Z","timestamp":1587513600000},"content-version":"vor","delay-in-days":0,"URL":"https:\/\/academic.oup.com\/journals\/pages\/open_access\/funder_policies\/chorus\/standard_publication_model"}],"funder":[{"name":"U.S. Office of the Assistant Secretary of Defense for Health Affairs"},{"name":"Technology\/Therapeutic Development Award of the Peer-Reviewed Medical Research Program","award":["PR181262"],"award-info":[{"award-number":["PR181262"]}]},{"name":"Technology\/Therapeutic Development Award of the Peer-Reviewed Medical Research Program","award":["W81XWH-19-1-0473"],"award-info":[{"award-number":["W81XWH-19-1-0473"]}]},{"DOI":"10.13039\/100000002","name":"NIH","doi-asserted-by":"publisher","award":["HL140197"],"award-info":[{"award-number":["HL140197"]}],"id":[{"id":"10.13039\/100000002","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/100000002","name":"NIH","doi-asserted-by":"publisher","award":["UH3 HL141798"],"award-info":[{"award-number":["UH3 HL141798"]}],"id":[{"id":"10.13039\/100000002","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/100010447","name":"DZHK","doi-asserted-by":"publisher","id":[{"id":"10.13039\/100010447","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/100010447","name":"German Centre for Cardiovascular Research","doi-asserted-by":"publisher","id":[{"id":"10.13039\/100010447","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/501100012680","name":"University Medical Center Hamburg-Eppendorf","doi-asserted-by":"publisher","id":[{"id":"10.13039\/501100012680","id-type":"DOI","asserted-by":"publisher"}]}],"content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":[],"published-print":{"date-parts":[[2020,7,15]]},"abstract":"<jats:title>Abstract<\/jats:title>\n               <jats:p>Inherited arrhythmias are disorders caused by one or more genetic mutations that increase the risk of arrhythmia, which result in life-long risk of sudden death. These mutations either primarily perturb electrophysiological homeostasis (e.g. long QT syndrome and catecholaminergic polymorphic ventricular tachycardia), cause structural disease that is closely associated with severe arrhythmias (e.g. hypertrophic cardiomyopathy), or cause a high propensity for arrhythmia in combination with altered myocardial structure and function (e.g. arrhythmogenic cardiomyopathy). Currently available therapies offer incomplete protection from arrhythmia and fail to alter disease progression. Recent studies suggest that gene therapies may provide potent, molecularly targeted options for at least a subset of inherited arrhythmias. Here, we provide an overview of gene therapy strategies, and review recent studies on gene therapies for catecholaminergic polymorphic ventricular tachycardia and hypertrophic cardiomyopathy caused by MYBPC3 mutations.<\/jats:p>","DOI":"10.1093\/cvr\/cvaa107","type":"journal-article","created":{"date-parts":[[2020,4,15]],"date-time":"2020-04-15T19:16:24Z","timestamp":1586978184000},"page":"1635-1650","source":"Crossref","is-referenced-by-count":44,"title":["Gene therapy for inherited arrhythmias"],"prefix":"10.1093","volume":"116","author":[{"ORCID":"https:\/\/orcid.org\/0000-0003-0825-6580","authenticated-orcid":false,"given":"Vassilios J","family":"Bezzerides","sequence":"first","affiliation":[{"name":"Department of Cardiology, Boston Children\u2019s Hospital, 300 Longwood Ave, Boston, MA 02115, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-6846-6435","authenticated-orcid":false,"given":"Maksymilian","family":"Prondzynski","sequence":"first","affiliation":[{"name":"Department of Cardiology, Boston Children\u2019s Hospital, 300 Longwood Ave, Boston, MA 02115, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-8776-5820","authenticated-orcid":false,"given":"Lucie","family":"Carrier","sequence":"first","affiliation":[{"name":"Institute of Experimental and Clinical Pharmacology and Toxicology, Cardiovascular Research Center, University Medical Center Hamburg-Eppendorf, Hamburg, Germany"},{"name":"DZHK (German Centre for Cardiovascular Research), partner site, Hamburg\/Kiel\/L\u00fcbeck, Hamburg, Germany"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-4551-8079","authenticated-orcid":false,"given":"William T","family":"Pu","sequence":"first","affiliation":[{"name":"Department of Cardiology, Boston Children\u2019s Hospital, 300 Longwood Ave, Boston, MA 02115, USA"},{"name":"Harvard Stem Cell Institute, 7 Divinity Avenue, Cambridge, MA 02138, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"286","published-online":{"date-parts":[[2020,4,22]]},"reference":[{"key":"2020070907341964800_cvaa107-B1","doi-asserted-by":"crossref","first-page":"160","DOI":"10.2174\/138920208784340768","article-title":"Molecular mechanisms of inherited arrhythmias","volume":"9","author":"Wolf","year":"2008","journal-title":"Curr Genomics"},{"key":"2020070907341964800_cvaa107-B2","doi-asserted-by":"crossref","first-page":"2441","DOI":"10.1056\/NEJMoa1510687","article-title":"A prospective study of sudden cardiac death among children and young adults","volume":"374","author":"Bagnall","year":"2016","journal-title":"N Engl J 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cardiomyopathy","volume":"62","author":"Goff","year":"2019","journal-title":"Prog Cardiovasc Dis"},{"key":"2020070907341964800_cvaa107-B6","doi-asserted-by":"crossref","first-page":"e73","DOI":"10.1016\/j.hrthm.2017.10.036","article-title":"2017 AHA\/ACC\/HRS guideline for management of patients with ventricular arrhythmias and the prevention of sudden cardiac death: a report of the American College of Cardiology\/American Heart Association Task Force on Clinical Practice Guidelines and the Heart Rhythm Society","volume":"15","author":"Al-Khatib","year":"2018","journal-title":"Heart Rhythm"},{"key":"2020070907341964800_cvaa107-B7","first-page":"e783","article-title":"2011 ACCF\/AHA guideline for the diagnosis and treatment of hypertrophic 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