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Lysosomal Storage Disorders Research / Virus-based Gene Therapy Research / CRISPR and Genetic Engineering · Journal article
Current Gene Therapy · July 17, 2026
A consensus or society position rather than new primary data.
This is a narrative review of genome editing technologies (CRISPR/Cas9, base editing, prime editing) in cardiovascular research and disease from 2020–2025. It documents proof-of-concept work in model organisms, mechanistic promise in atherosclerosis and cardiomyopathy, and early-stage clinical feasibility in transthyretin amyloidosis, while identifying delivery, off-target effects, and ethical barriers as unsolved challenges to clinical translation.
Narrative literature review. Peer-reviewed literature on genome editing in cardiovascular biology and disease. Intervention: Review of genome editing technologies and their cardiovascular applications.
Base editing enables precise single-nucleotide corrections, particularly PCSK9 targeting with long-lasting reductions in LDL cholesterol levels in humans Prime editing extends capability to complex mutations, including RBM20 in dilated cardiomyopathy Early-stage clinical trials targeting transthyretin amyloidosis demonstrate feasibility of in vivo genome editing
Second-generation cardiotropic AAV vectors and lipid nanoparticles continue to improve cardiac delivery and safety profiles
The source did not state who this applies to in practice.
A narrative review synthesizing peer-reviewed literature on genome editing applications in cardiovascular disease, offering a landscape assessment and discussion of tools, mechanisms, and remaining challenges rather than reporting original trial data or definitive clinical evidence.
As stated by the source record.
Graded across the dimensions that decide whether you should act, each from what the source actually supports. There is no single score, and where a dimension was not assessed it says so.
What is missing. This record has no reported figures. That is a gap in the analysis, not a judgement about the study.
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.
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