title: Gene therapy for the treatment of heart failure: promise postponed.
abstract: Gene therapy has emerged as a powerful tool in targeting the molecular mechanisms implicated in heart failure. Refinements in vector technology, including the development of recombinant adeno-associated vectors, have allowed for safe, long-term, and efficient gene transfer to the myocardium. These advancements, coupled with evolving delivery techniques, have placed gene therapy as a viable therapeutic option for patients with heart failure. However, after much promise in early-phase clinical trials, the more recent larger clinical trials have shown disappointing results, thus forcing the field to re-evaluate current vectors, delivery systems, targets, and endpoints. We provide here an updated review of current cardiac gene therapy programmes that have been or are being translated into clinical trials.
authors.affiliation: Icahn School of Medicine at Mount Sinai, One Gustave L. Levy Place, Box 1030, New York, NY 10029-6574, USA Sorbonne Universités, UPMC University Paris 06, AP-HP, Institute of Cardiometabolism and Nutrition (ICAN), Pitié-Salpêtrière Hospital, F-75013 Paris, France.
authors.forename: Jean-Sebastien
authors.initials: JS
authors.lastname: Hulot
authors.affiliation: Icahn School of Medicine at Mount Sinai, One Gustave L. Levy Place, Box 1030, New York, NY 10029-6574, USA.
authors.forename: Kiyotake
authors.initials: K
authors.lastname: Ishikawa
authors.affiliation: Icahn School of Medicine at Mount Sinai, One Gustave L. Levy Place, Box 1030, New York, NY 10029-6574, USA roger.hajjar@mssm.edu.
authors.forename: Roger J
authors.initials: RJ
authors.lastname: Hajjar
mesh_terms: D043925:Angiogenesis Inducing Agents / Q000627:therapeutic use; D000818:Animals; D002118:Calcium / Q000378:metabolism; D015220:Calcium Channels / Q000378:metabolism; D054377:Chemokine CXCL12 / Q000235:genetics; D002986:Clinical Trials as Topic; D018014:Gene Transfer Techniques; D015316:Genetic Therapy / Q000379:methods / Q000639:trends*; D005822:Genetic Vectors; D006333:Heart Failure / Q000503:physiopathology / Q000628:therapy*; D006801:Humans; D051379:Mice; D009200:Myocardial Contraction / Q000502:physiology; D053498:Sarcoplasmic Reticulum Calcium-Transporting ATPases / Q000235:genetics; D013234:Stem Cells / Q000502:physiology; D042461:Vascular Endothelial Growth Factor A / Q000627:therapeutic use
chemical_list: D043925:Angiogenesis Inducing Agents; C515255:CXCL12 protein, human; D015220:Calcium Channels; D054377:Chemokine CXCL12; D042461:Vascular Endothelial Growth Factor A; D053498:Sarcoplasmic Reticulum Calcium-Transporting ATPases; D002118:Calcium
keywords: Adeno-associated vectors; Excitation–contraction coupling; Gene therapy; Heart failure; Sarcoplasmic reticulum calcium ATPase