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Adeno-Associated Virus Vector Mediated Gene Therapy: A Promising Approach to Transform Hypertrophic Cardiomyopathy Treatment
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DOI：10.1002/biot.70262.png)
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Abstract
En 中文
Hypertrophic cardiomyopathy (HCM) is a genetically heterogeneous myocardial disease that usually arises from mutations in key sarcomeric proteins, particularly MYBPC3 and MYH7. Current pharmacological treatments, such as myosin inhibitors, calcium channel blockers, and β-blockers, 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-associated virus (AAV) vectors, particularly serotype AAV9, demonstrate strong cardiac tropism, long-term expression, and favorable safety, making them ideal for cardiac gene delivery. AAV-mediated gene therapy has the potential to represent paradigm shift in HCM treatment, transitioning from symptomatic management to curative interventions. However, challenges such as long-term 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-mediated 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-based therapeutic strategies for cardiomyopathy, preclinical findings, and emerging clinical trials of AAV9-mediated gene therapy.
Keywords:
AAV vector
gene therapy
hypertrophic cardiomyopathy
vector serotype
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