Adeno-associated virus vectors demonstrate strong potential for cardiac gene therapy in preclinical models, but clinical translation is currently limited by immune responses and inadequate myocardial delivery.
AAV viral vectors, especially AAV9, represent a promising gene therapy platform for cardiac disorders, but require further engineering to overcome immune and delivery barriers for successful clinical translation.
Ischemic and non-ischemic cardiac diseases including arrhythmogenic cardiomyopathy and myocardial infarction, remain one of the leading causes of death worldwide despite significant advances in cardiovascular therapeutics. Current treatment strategies such as β-blockers, angiotensin-converting-enzyme inhibitors, and cardiac surgical interventions that include implantations of pacemakers and cardioverter-defibrillators are effective but often associated with serious side effects. In recent years, multiple cell-based therapies have emerged, aiming either to regeneration of myocardial or myofascial tissues or to correct defective gene using gene-transfer tools. Adeno-associated virus (AAVs), initially identified as contaminants of adeno-virus preparations, have since become one of the most important viral vectors for gene-transfer, especially in mammalian cells. This review analyzes and summarizes various AAV serotypes utilized in gene therapy programs for preclinical and clinical assays for cardiac disease.
Kumar et al. (Thu,) conducted a review in Inherited and acquired cardiac diseases. Adeno-associated virus (AAV) vectors was evaluated. Adeno-associated virus vectors demonstrate strong potential for cardiac gene therapy in preclinical models, but clinical translation is currently limited by immune responses and inadequate myocardial delivery.
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