Abstract :

CRISPR-Cas systems have revolutionized gene editing, offering precise and efficient genome modifications with vast applications in biomedical research and therapeutic interventions. This technology has surpassed traditional methods such as zinc-finger nucleases (ZFNs) and transcription activator-like effector nucleases (TALENs) due to its simplicity, cost-effectiveness, and high accuracy. In cardiovascular disease (CVD) research, CRISPR has been instrumental in generating precise disease models, identifying genetic risk factors, and developing potential therapeutic strategies. Genome editing has enabled the correction of monogenic mutations linked to inherited cardiomyopathies and arrhythmias while also targeting metabolic risk factors such as PCSK9 to reduce atherosclerosis.

Advancements such as base editing, prime editing, and RNA editing have further enhanced CRISPR’s precision, minimizing off-target effects and improving therapeutic applicability. Delivery methods, including adeno-associated viral (AAV) vectors and nanoparticle-based strategies, have been optimized to enhance efficiency and safety. Despite these breakthroughs, challenges such as unintended genetic modifications, immune responses, and ethical concerns regarding germline editing persist. The successful clinical translation of CRISPR therapies will require further refinement, rigorous validation, and regulatory oversight to ensure safety and efficacy.

As research continues to advance, CRISPR-based genome editing holds immense promise for transforming cardiovascular medicine. Future developments in delivery technologies, improved gene-editing accuracy, and ethical considerations will play a crucial role in integrating CRISPR into clinical practice. With continued innovation, CRISPR has the potential to become a groundbreaking therapeutic tool, offering precise and long-lasting solutions for cardiovascular diseases and other genetic disorders.

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Keywords :

Cardiovascular diseases, CRISPR-Cas, gene therapy, Genome Editing, Precision Medicine.

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References :

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