title: Gene therapy for heart failure.
abstract: Congestive heart failure accounts for half a million deaths per year in the United States. Despite its place among the leading causes of morbidity, pharmacological and mechanic remedies have only been able to slow the progression of the disease. Today's science has yet to provide a cure, and there are few therapeutic modalities available for patients with advanced heart failure. There is a critical need to explore new therapeutic approaches in heart failure, and gene therapy has emerged as a viable alternative. Recent advances in understanding of the molecular basis of myocardial dysfunction, together with the evolution of increasingly efficient gene transfer technology, have placed heart failure within reach of gene-based therapy. The recent successful and safe completion of a phase 2 trial targeting the sarcoplasmic reticulum calcium ATPase pump (SERCA2a), along with the start of more recent phase 1 trials, opens a new era for gene therapy for the treatment of heart failure.
authors.affiliation: Cardiovascular Research Center, Mount Sinai Medical Center, New York, NY 10029, USA.
authors.forename: Lisa
authors.initials: L
authors.lastname: Tilemann
authors.forename: Kiyotake
authors.initials: K
authors.lastname: Ishikawa
authors.forename: Thomas
authors.initials: T
authors.lastname: Weber
authors.forename: Roger J
authors.initials: RJ
authors.lastname: Hajjar
mesh_terms: D000818:Animals; D004195:Disease Models, Animal; D004285:Dogs; D018014:Gene Transfer Techniques; D015316:Genetic Therapy; D006321:Heart / Q000503:physiopathology; D006331:Heart Diseases / Q000503:physiopathology / Q000628:therapy; D006333:Heart Failure / Q000503:physiopathology / Q000628:therapy*; D006801:Humans; D051379:Mice; D051381:Rats; D053498:Sarcoplasmic Reticulum Calcium-Transporting ATPases / Q000502:physiology
chemical_list: D053498:Sarcoplasmic Reticulum Calcium-Transporting ATPases