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Current pharmacological treatments, such as myosin inhibitors, calcium channel blockers, and \u03b2\u2010blockers, reduce symptoms but do not address the underlying genetic cause of the illness. Gene therapy has emerged as a promising strategy to address HCM. Adeno\u2010associated virus (AAV) vectors, particularly serotype AAV9, demonstrate strong cardiac tropism, long\u2010term expression, and favorable safety, making them ideal for cardiac gene delivery. AAV\u2010mediated gene therapy has the potential to represent paradigm shift in HCM treatment, transitioning from symptomatic management to curative interventions. However, challenges such as long\u2010term safety, vector immunogenicity, treatment durability, and scalable manufacturing must be addressed before it can be widely adopted in routine clinical practice. The present review explores the potential of AAV\u2010mediated gene therapy in the treatment of hypertrophic cardiomyopathy. The review discusses AAV vectors, focusing on capsid structure, their genome organization, and cardiotropic serotypes optimized for cardiovascular gene delivery. The article explores how different AAV serotypes, particularly AAV9, transduce cardiomyocytes and achieve efficient, selective cardiac tropism. Furthermore, the article discusses AAV\u2010based therapeutic strategies for cardiomyopathy, preclinical findings, and emerging clinical trials of AAV9\u2010mediated gene therapy.<\/jats:p>","DOI":"10.1002\/biot.70262","type":"journal-article","created":{"date-parts":[[2026,6,17]],"date-time":"2026-06-17T11:43:30Z","timestamp":1781696610000},"source":"Crossref","is-referenced-by-count":0,"title":["Adeno\u2010Associated Virus Vector Mediated Gene Therapy: A Promising Approach to Transform Hypertrophic Cardiomyopathy Treatment"],"prefix":"10.1002","volume":"21","author":[{"ORCID":"https:\/\/orcid.org\/0009-0005-7860-0094","authenticated-orcid":false,"given":"Saif","family":"Siddiqui","sequence":"first","affiliation":[{"name":"Department of Pharmacology SVKM's Dr. Bhanuben Nanavati College of Pharmacy  Mumbai India"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Siddhi Bagwe","family":"Parab","sequence":"additional","affiliation":[{"name":"Department of Pharmacology SVKM's Dr. Bhanuben Nanavati College of Pharmacy  Mumbai India"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-4302-9300","authenticated-orcid":false,"given":"Lokesh Kumar","family":"Bhatt","sequence":"additional","affiliation":[{"name":"Department of Pharmacology SVKM's Dr. Bhanuben Nanavati College of Pharmacy  Mumbai India"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"311","published-online":{"date-parts":[[2026,6,17]]},"reference":[{"key":"e_1_2_15_2_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.jacc.2023.10.032"},{"key":"e_1_2_15_3_1","doi-asserted-by":"publisher","DOI":"10.3390\/ijms22168933"},{"key":"e_1_2_15_4_1","doi-asserted-by":"publisher","DOI":"10.1161\/CIRCRESAHA.117.311059"},{"key":"e_1_2_15_5_1","doi-asserted-by":"publisher","DOI":"10.21037\/cdt.2019.02.01"},{"key":"e_1_2_15_6_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.jchf.2018.02.010"},{"key":"e_1_2_15_7_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.ijcard.2024.132913"},{"key":"e_1_2_15_8_1","doi-asserted-by":"publisher","DOI":"10.1161\/JAHA.124.037639"},{"key":"e_1_2_15_9_1","doi-asserted-by":"publisher","DOI":"10.1186\/1743\u2010422X\u20104\u201099"},{"key":"e_1_2_15_10_1","doi-asserted-by":"publisher","DOI":"10.1038\/mt.2008.90"},{"key":"e_1_2_15_11_1","doi-asserted-by":"publisher","DOI":"10.1126\/scitranslmed.3002097"},{"key":"e_1_2_15_12_1","doi-asserted-by":"publisher","DOI":"10.1111\/j.1752\u20108062.2010.00190.x"},{"key":"e_1_2_15_13_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.ymthe.2022.07.003"},{"key":"e_1_2_15_14_1","doi-asserted-by":"publisher","DOI":"10.1161\/CIRCRESAHA.111.252981"},{"key":"e_1_2_15_15_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.cardiores.2005.12.017"},{"key":"e_1_2_15_16_1","doi-asserted-by":"publisher","DOI":"10.3390\/pathogens12111301"},{"key":"e_1_2_15_17_1","doi-asserted-by":"publisher","DOI":"10.1161\/CIRCGEN.122.003730"},{"key":"e_1_2_15_18_1","doi-asserted-by":"publisher","DOI":"10.1038\/s41434\u2010025\u201000553\u20106"},{"key":"e_1_2_15_19_1","doi-asserted-by":"publisher","DOI":"10.1161\/hy0202.103472"},{"key":"e_1_2_15_20_1","doi-asserted-by":"publisher","DOI":"10.3389\/fimmu.2022.975803"},{"key":"e_1_2_15_21_1","doi-asserted-by":"publisher","DOI":"10.3390\/ijms27073196"},{"key":"e_1_2_15_22_1","doi-asserted-by":"publisher","DOI":"10.3390\/pathogens12111301"},{"key":"e_1_2_15_23_1","doi-asserted-by":"publisher","DOI":"10.1038\/s41467\u2010025\u201057481\u20107"},{"key":"e_1_2_15_24_1","doi-asserted-by":"publisher","DOI":"10.1097\/HCO.0000000000000386"},{"key":"e_1_2_15_25_1","doi-asserted-by":"publisher","DOI":"10.1093\/cvr\/cvaf200"},{"key":"e_1_2_15_26_1","doi-asserted-by":"publisher","DOI":"10.1172\/jci.insight.130182"},{"key":"e_1_2_15_27_1","first-page":"49","article-title":"Viral Expression Cassette Elements to Enhance Transgene Target Specificity and Expression in Gene Therapy","volume":"19","author":"Powell S. 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