Gene/Genome Editing in Cardiovascular Biology and Disease.
TL;DR
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.
Abstract
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.