Cardiology in reviewOct 22, 2025

Using Gene Editing to Fix Inherited Ion Channel Disorders

CRISPR Gene Editing Weekly Brief ↗PubMed ↗DOI ↗

Updated Jan 3, 2026

Abstract

Inherited cardiac channelopathies contribute to significant arrhythmic morbidity and sudden cardiac death in young individuals.

Current therapies can reduce risk but do not address the underlying genetic causes of these conditions.
CRISPR/Cas9 genome editing has the potential to directly repair disease-causing genetic variants.
Recent advances allow for precise correction of mutations in preclinical models, demonstrating promising results.
Animal models and patient-derived heart cells show restoration of electrical stability and suppression of arrhythmias.
Challenges such as off-target effects and delivery barriers need to be addressed for effective clinical application.
Innovations like improved delivery methods and artificial intelligence may aid in the future of gene editing for these disorders.

Simplified

Inherited cardiac channelopathies, including long QT syndrome, Brugada syndrome, and catecholaminergic polymorphic ventricular tachycardia, are major causes of arrhythmic morbidity and sudden cardiac death in young individuals. Current therapies, such as pharmacologic agents, implantable cardioverter-defibrillators, and lifestyle modifications, reduce risk but fail to correct the underlying genetic substrate, creating an urgent need for curative strategies. CRISPR/Cas9 genome editing has emerged as a transformative platform with the potential to directly repair pathogenic variants. Recent advances in base and prime editing, together with novel viral and nonviral delivery platforms, have enabled precise correction of disease-causing mutations in preclinical models. Proof-of-concept studies using animal models and patient-derived iPSC-cardiomyocytes demonstrate restoration of electrophysiologic stability, suppression of arrhythmias, and durable functional benefit. Nevertheless, translational challenges remain, including off-target effects, delivery barriers, immune responses, scalability, and ethical considerations. Ongoing innovations-such as engineered nucleases, improved delivery vectors, immunogenicity mitigation strategies, and integration of artificial intelligence for personalized guide design-are expected to accelerate clinical translation. This review synthesizes current knowledge on CRISPR-based strategies for inherited channelopathies, highlighting both the promise and limitations of gene editing as a path toward durable, disease-modifying therapies capable of preventing sudden cardiac death.

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Competing interests

Disclosure: The authors declare no conflicts of interest.

PubMed

Using Gene Editing to Fix Inherited Ion Channel Disorders

Abstract

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Abstract

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Related papers

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

What Lands in Your Inbox Each Week:

Recent issues from the crispr gene editing brief