Top stories on gene therapy for genetic heart disease (2024)

Gene therapy is defined as the modification of gene expression via the introduction of therapeutic nucleic acid with the intention to treat or prevent disease. Mechanistically, gene therapy strategies aim to (1) replace/supplement a defective gene (eg, loss-of-function [LOF] disorders caused by haploinsufficiency) with a functional copy; (2) suppress/silence a defective gene (eg, gain-of-function [GOF] disorders); (3) suppress the normal and mutant allele and replace with normal gene in a hybrid manner (eg, for dominant negative LOF disorders); or (4) directly edit the genome to remove a disease-causative genetic lesion.
Source: Heart Rhythm - Category: Cardiology Authors: Tags: Top Stories: Clinical Electrophysiology Source Type: research