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Updated: Jul 21, 2025

Gene Transfer for Ischemic Heart Failure in a Preclinical Model
Published on: May 15, 2011
Gene therapy for heart failure and cardiomyopathies
Alessia Argirò1, Jeffrey Ding2, Eric Adler2
1Cardiomyopathy Unit, Careggi University Hospital, Florence, Italy.
Abstract:
Gene therapy strategies encompass a range of approaches, including gene replacement and gene editing. Gene replacement involves providing a functional copy of a modified gene, while gene editing allows for the correction of existing genetic mutations. Gene therapy has already received approval for treating genetic disorders like Leber's congenital amaurosis and spinal muscular atrophy. Currently, research is being conducted to explore its potential use in cardiology. This review aims to summarize the mechanisms behind different gene therapy strategies, the available delivery systems, the primary risks associated with gene therapy, ongoing clinical trials, and future targets, with a particular emphasis on cardiomyopathies.
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