Emerging CRISPR Therapies for Precision Gene Editing and Modulation in the Cardiovascular Clinic.

Document Type

Article

Publication Date

11-1-2024

Keywords

JGM, Humans, Gene Editing, Cardiovascular Diseases, CRISPR-Cas Systems, Genetic Therapy

JAX Source

Curr Cardiol Rep. 2024;26(11):1231-40.

ISSN

1534-3170

PMID

39287778

DOI

https://doi.org/10.1007/s11886-024-02125-3

Abstract

PURPOSE OF REVIEW: Outline the growing suite of novel genome editing tools powered by CRISPR-Cas9 technology that are rapidly advancing towards the clinic for the treatment of cardiovascular disorders.

RECENT FINDINGS: A diversity of new genome editors and modulators are being developed for therapies across myriad human diseases. Recent breakthroughs have improved the efficacy, safety, specificity, and delivery of CRISPR-mediated therapies that could impact heart disease in the next decade, though several challenges remain. Many iterations of the original CRISPR system have been developed seeking to leverage its vast therapeutic potential. As examples, nuclease-free editing, precision single-nucleotide editing, gene expression regulation, and epigenomic modifications are now feasible with the current CRISPR-mediated suite of enzymes. These emerging tools will be indispensable for the development of novel cardiovascular therapeutics as demonstrated by recent successes in both basic research laboratories and pre-clinical models. Here, we provide an overview of current and emerging CRISPR-mediated technologies as they pertain to the cardiovascular system, highlighting successful implementations and future challenges.

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