Heart (British Cardiac Society)Oct 20, 2025

RNA and gene therapies for heart disease: medical uses, delivery challenges, and new precision tools

CRISPR Gene Editing Weekly Brief ↗PubMed ↗DOI ↗

Updated Jan 3, 2026

Abstract

Recent approvals of RNA therapies such as patisiran, vutrisiran, and inclisiran demonstrate the clinical potential of siRNAs in treating cardiovascular diseases.

RNA and gene therapies are emerging as alternatives to traditional cardiovascular treatments, addressing issues of efficacy and patient adherence.
RNA-based therapeutics, including small-interfering RNAs and antisense oligonucleotides, target specific molecular pathways in the liver.
CRISPR-Cas9 technologies may provide permanent correction of genetic mutations associated with hereditary cardiovascular conditions.
Challenges remain in effectively delivering these therapies to cardiac tissues and optimizing their delivery systems.
Ongoing phase I clinical trials are exploring the potential of these advanced therapies in the field of cardiovascular medicine.

Simplified

Cardiovascular diseases remain a leading cause of global mortality despite advancements in pharmacotherapies, with current treatments facing challenges related to efficacy, tolerability and patient adherence. In response, advanced therapies, such as RNA and gene therapies, have emerged as a promising alternative for addressing both acquired and monogenic cardiovascular conditions. This review explores the current landscape of RNA and gene therapies for cardiovascular disease, focusing on RNA-based therapeutics such as small-interfering RNAs (siRNAs), antisense oligonucleotides and clustered regularly interspaced short palindromic repeats and associated Cas9 endonuclease (CRISPR-Cas9)-based gene editing systems. Recent European Medicines Agency and Food and Drug Administration-approved RNA therapies, including patisiran, vutrisiran and inclisiran, which employ lipid nanoparticle delivery systems, highlight the clinical potential of siRNAs for targeting hepatic molecular pathways. Emerging CRISPR-Cas9 technologies are poised to address genetic mutations at their source, offering permanent correction of pathogenic variants and the potential to treat a broad range of hereditary cardiovascular conditions. Together, these therapies represent a major leap forward in precision medicine, offering long-lasting therapeutic effects and improved patient care and adherence. However, many challenges remain, particularly in targeting such therapies to cardiac tissues and optimising delivery systems. This review discusses the current state of the art in cardiovascular RNA and gene therapies, including current evidence, delivery challenges and the current landscape of gene and RNA therapies in phase I clinical trials and beyond.

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Competing interests

Competing interests: None declared.

PubMed

RNA and gene therapies for heart disease: medical uses, delivery challenges, and new precision tools

Abstract

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Funding

Competing interests

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Abstract

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Funding

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Related papers

You found one interesting study. We’ll send the next 7.

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