title: Gene therapy in heart failure. SERCA2a as a therapeutic target.
abstract: The treatment of heart failure (HF) may be entering a new era with clinical trials currently assessing the value of gene therapy as a novel therapeutic strategy. If these trials demonstrate efficacy then a new avenue of potential treatments could become available to the clinicians treating HF. In principle, gene therapy allows us to directly target the underlying molecular abnormalities seen in the failing myocyte. In this review we discuss the fundamentals of gene therapy and the challenges of delivering it to patients with HF. The molecular abnormalities underlying HF are discussed along with potential targets for gene therapy, focusing on SERCA2a. We discuss the laboratory and early clinical evidence for the benefit of SERCA2a gene therapy in HF. Finally, we discuss the ongoing clinical trials of SERCA2a gene therapy and possible future directions for this treatment.
authors.affiliation: NIHR Cardiovascular Biomedical Research Unit, Royal Brompton Hospital.
authors.forename: Carl
authors.initials: C
authors.lastname: Hayward
authors.forename: Hitesh
authors.initials: H
authors.lastname: Patel
authors.forename: Alexander
authors.initials: A
authors.lastname: Lyon
mesh_terms: D000818:Animals; D002986:Clinical Trials as Topic; D015316:Genetic Therapy; D006333:Heart Failure / Q000235:genetics / Q000378:metabolism / Q000473:pathology / Q000628:therapy*; D006801:Humans; D032383:Myocytes, Cardiac / Q000378:metabolism* / Q000473:pathology; D053498:Sarcoplasmic Reticulum Calcium-Transporting ATPases / Q000096:biosynthesis* / Q000235:genetics
chemical_list: D053498:Sarcoplasmic Reticulum Calcium-Transporting ATPases