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

Gene/Genome Editing in Cardiovascular Biology and Disease.

INTRODUCTION Cardiovascular diseases continue to be the leading cause of death worldwide. Traditional medicines relieve symptoms and slow the advancement of the disease, but fail to fix genetic problems at their roots. Cardiovascular science has dramatically changed thanks to advancements in genome editing tools, such as CRISPR/Cas9 and its successor technologies. These types of genome editing tools enable researchers and physicians to precisely and programmatically manipulate specific genetic loci related to cardiovascular diseases, offering hope for developing new curative therapies. METHODS This article is based on a complete review of peer-reviewed literature published between 2020 and 2025. A systematic search of databases (PubMed, Web of Science, Scopus, and others) for literature using the keywords (genome editing; CRISPR/Cas9; base editing; prime editing; cardiovascular disease; cardiomyopathy; atherosclerosis; etc.) was completed. RESULTS CRISPR/Cas9 makes it easy and fast to create genetically modified cardiac model organisms to evaluate pathogenic variation. Using base editing is an effective way to perform precise single- nucleotide corrections, particularly with respect to PCSK9 targeting and its association with long-lasting reductions in LDL cholesterol levels in humans. Prime editing extends this capability to complex mutations, including RBM20 in dilated cardiomyopathy. Early-stage clinical trials targeting transthyretin amyloidosis demonstrate the feasibility of in vivo genome editing. Secondgeneration cardiotropic AAV vectors and lipid nanoparticles continue to improve cardiac delivery and safety profiles. DISCUSSION Genome editing shifted cardiovascular research from associative genetics toward causal intervention. Next-generation editors reduce double-strand break-associated risks, enhancing clinical suitability. Still remaining challenges include efficient delivery in a tissue-specific manner, off-target effects, immunity, and ethical considerations related to permanent genomic modification. CONCLUSION Genome editing is a paradigm-shifting development within the field of cardiology that promises a long-term genetic remedy. Yet further optimization and development within genome editing and its guidelines will be important for making such a paradigm shift successful.

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

Advances in Clinical Research on CRISPR-Cas9 for the Treatment of Common Monogenic Diseases

The success of CRISPR-Cas9 in monogenic diseases represents the prelude to precision medicine, whereas conquering complex diseases will require systematic leaps in target discovery, delivery technology, and safety profiles.

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 Therapies in Treating Monogenic Neurodevelopmental Disorders

This review evaluates the potential of CRISPR-based editing as a therapeutic strategy for monogenic NDDs and evaluates the limitations that must be addressed before its widespread application in human patients.

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.