Gene/Genome Editing in Cardiovascular Biology and Disease.

Recent applications of CRISPR-Cas systems in animal models of human cardiovascular disease: Review article

A review of studies in which the creation of a CRISPR-Cas9 animal model has been used to find genetic drivers and develop therapies across 4 CVD domains shows the vast range of gene editing applications and the value of models that parallel human genetic pathophysiology for specific disease processes.

Precision Genome Editing Strategies for Enduring Lipid Lowering in Atherosclerosis.

Atherosclerosis continues to be a primary contributor to global cardiovascular mortality, influenced by intricate lipid and inflammatory mechanisms. Despite the efficacy of conventional pharmacotherapies, ongoing issues of patient non-adherence and residual risk have prompted the exploration of more enduring therapeutic alternatives. This review examines the significant transition in cardiovascular research from conventional, wide knockout models to the utilization of advanced precision genome editing methods, particularly emphasizing CRISPR-Cas9, base editing, and prime editing. These sophisticated molecular tools allow for the accurate insertion and rectification of single-nucleotide variants without causing double-strand breaks, marking a significant shift from rudimentary gene disruption to precise variant engineering. By specifically targeting essential lipid-regulating genes like proprotein convertase subtilisin/kexin type 9 (PCSK9) and angiopoietin-like 3 (ANGPTL3), precision editing presents an exceptional opportunity for lasting, one-shot lipid-lowering treatments. Additionally, we examine the advancement of preclinical modeling, emphasizing humanized models that precisely represent population genetics. This review highlights the essential obstacles to clinical translation, focusing on the optimization of delivery systems such as adeno-associated viruses (AAVs) and lipid nanoparticles (LNPs), as well as the thorough assessment of off-target effects and ethical implications.

The Vascular Genome as a Therapeutic Target: A Systematic Review of CRISPR-based Gene Editing In Vascular Disease

Targeting the Hallmarks of Ageing: Pharmacological Challenges and Breakthroughs in CRISPR Delivery Systems and Future Prospects.

CRISPR has emerged as a next-generation gene-editing tool with the potential to target the molecular pathways associated with ageing and related disorders. It functions through RNA-guided Cas nucleases, directing DNA cleavage and utilizing the native DNA repair machinery for genetic manipulations. Advances in CRISPR technology have significantly enhanced the precision and flexibility of techniques for genome editing. The enzyme Cas9's ability to cut DNA at exact site has revolutionized genome editing by enabling accurate modifications within living eukaryotic cells. This review critically examines recent developments in CRISPR-based technologies, including Cas9, Cas12, base editing, prime editing, and CRISPR-mediated gene regulation. It highlights their rising applications in ageing research, with more emphasis on neurodegenerative disorders such as Alzheimer's and Parkinson's diseases. The review also discusses the major pharmacological and translational challenges that currently limit clinical applications, including inefficient tissue-specific delivery, off-target genome editing, immunogenicity, manufacturing complexity, and long-term safety concerns. Also, recent progress in both, viral and non-viral delivery methods are critically evaluated, including adeno-associated viruses, lentivirus vectors, lipid nanoparticles, gold nanoparticles, exosomes, electroporation, and microinjection, is thoroughly discussed to highlight their therapeutic potential and translational limitations. Current studies indicate that CRISPR-based approaches have preclinical potential for targeting important hallmarks of ageing, particularly genomic instability, telomere attrition, and mitochondrial dysfunction. Other hallmarks of ageing, such as stem cell exhaustion, epigenetic modifications, and microbiome changes, are at earlier stages of development. Overall, this review describes future strategies for developing safe, precise, and clinically translatable CRISPR-based treatments to promote healthy ageing.

CRISPR-Based Gene Editing in Human and Animal Health: Revolutionizing Disease Resistance and Treatment

CRISPR gene-editing technology has revolutionized modern genetics, offering precise and efficient modifications across multiple domains, including human medicine, agriculture, and veterinary science. This study explores the diverse applications of CRISPR, highlighting its role in treating genetic disorders such as sickle cell anemia and Duchenne muscular dystrophy, advancing cancer immunotherapies, and developing CRISPR-based antiviral therapies for HIV and COVID-19. In agriculture, CRISPR has facilitated the development of disease-resistant livestock, enhanced crop yields, and improved food sustainability. Additionally, CRISPR is being integrated with artificial intelligence (AI) and bioinformatics to optimize gene-editing accuracy, predict off-target effects, and accelerate drug discovery. Despite these advancements, CRISPR faces significant challenges, including ethical dilemmas surrounding germline editing, regulatory inconsistencies across countries, high costs of gene therapies, and concerns about genetic inequality. The legal and social implications of CRISPR remain complex, requiring global cooperation to establish standardized regulations and ensure equitable access to genetic therapies. Emerging innovations such as base editing, prime editing, and epigenetic modifications offer promising solutions to improve CRISPR’s precision and safety. Looking ahead, CRISPR’s long-term success will depend on responsible scientific advancements, ethical oversight, and public acceptance. With the continued refinement of gene-editing techniques and AI-driven CRISPR optimizations, this technology holds the potential to revolutionize medicine, agriculture, and environmental conservation. However, careful implementation and transparent discussions are essential to navigate the ethical, legal, and societal challenges that accompany CRISPR’s rapid development.

CRISPR-Based Genome Editing in Contemporary Clinical Medicine: Therapeutic Translation, Global Challenges and the Future of Precision Medicine

CRISPR has progressed from an experimental genome-engineering technology to a clinically relevant therapeutic platform, although its future impact will depend on the ability to combine molecular precision and durable therapeutic benefit with rigorous safety assessment, responsible governance, and equitable access across diverse populations and healthcare systems.