Strategies and advances in gene therapy for inherited cardiac diseases
XU Lin
TANG Kai
SHEN Li-shui
Abstract：Inherited cardiac diseases,caused by single or multiple gene mutations,represent a category of cardiovascular disorders characterized by sudden cardiac death and heart failure as primary clinical manifestations.Existing treatments struggle to reverse their pathophysiological progression.Gene therapy offers a groundbreaking strategy for eradicating these diseases through targeted correction of pathogenic mutations or regulation of aberrant gene expression.This article reviews recent advances in gene therapy for inherited cardiac disorders,discusses the advantages,limitations,and optimization approaches of different delivery vectors and molecular strategies,and summarizes their potential applications in inherited arrhythmias and cardiomyopathies.Additionally,it outlines the latest translational pathways addressing challenges such as balancing delivery efficiency-specificity and overcoming limitations of animal models.
Keywords：Inherited cardiac diseasesGene therapyAdeno-associated virusesGene editing
Publication Date：2025-10-20
Online Publishing Date：2025-10-30(First online date of this platform, not the publication date of the document)
Pages：6( 877-882 )