GENE THERAPY: POTENTIAL FOR TREATING CARDIOVASCULAR DISEASES AT THE DNA LEVEL
Keywords:
Gene therapy, cardiovascular diseases, CRISPR/Cas9, RNA-based therapy, heart failure, inherited cardiomyopathy, atherosclerosis, gene editing, viral vectors, lipid nanoparticles.Abstract
Cardiovascular diseases (CVDs) remain the leading cause of morbidity and mortality worldwide, necessitating novel therapeutic approaches beyond conventional treatments. Gene therapy has emerged as a promising strategy by targeting the underlying genetic causes of these diseases, offering potential long-term or even curative solutions. Recent advancements in gene delivery systems (AAVs, lentiviruses, lipid nanoparticles), genome editing technologies (CRISPR/Cas9, base editing, prime editing), and RNA-based therapies (siRNA, antisense oligonucleotides, mRNA therapies) have demonstrated significant potential in preclinical and clinical studies. This article explores the current methodologies of gene therapy in cardiology, including its application in heart failure, inherited cardiomyopathies, and atherosclerosis. Key findings include the use of cBIN1 and SERCA2a gene therapy to restore cardiac function, CRISPR-mediated correction of MYBPC3 mutations in hypertrophic cardiomyopathy, and PCSK9-targeted RNA therapies for lipid regulation. Despite promising outcomes, challenges such as efficient gene delivery, immune responses, long-term safety, and ethical concerns remain critical barriers to widespread clinical implementation. Ongoing research is focused on refining non-viral delivery systems, improving precision genome editing, and developing personalized gene therapy approaches. With continued advancements, gene therapy holds the potential to revolutionize cardiovascular medicine, shifting treatment paradigms from symptom management to molecular-level cures.
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