title: Potential of gene therapy as a treatment for heart failure.
abstract: Advances in understanding the molecular basis of myocardial dysfunction, together with the evolution of increasingly efficient gene transfer technology, make gene-based therapy a promising treatment option for heart conditions. Cardiovascular gene therapy has benefitted from recent advancements in vector technology, design, and delivery modalities. There is a critical need to explore new therapeutic approaches in heart failure, and gene therapy has emerged as a viable alternative. Advances in understanding of the molecular basis of myocardial dysfunction, together with the development of increasingly efficient gene transfer technology, has placed heart failure within reach of gene-based therapy. The recent successful and safe completion of a phase 2 trial targeting the cardiac sarcoplasmic/endoplasmic reticulum Ca2+ ATPase pump (SERCA2a) has the potential to open a new era for gene therapy for heart failure.
authors.affiliation: Cardiovascular Research Center, Mount Sinai School of Medicine, One Gustave Levy Place, Box 1030, New York, New York 10029, USA. roger.hajjar@mssm.edu
authors.forename: Roger J
authors.initials: RJ
authors.lastname: Hajjar
mesh_terms: D018014:Gene Transfer Techniques; D015316:Genetic Therapy / Q000379:methods*; D006333:Heart Failure / Q000235:genetics / Q000378:metabolism / Q000473:pathology / Q000628:therapy*; D009124:Muscle Proteins / Q000096:biosynthesis / Q000235:genetics*; D009206:Myocardium / Q000378:metabolism / Q000473:pathology; D053498:Sarcoplasmic Reticulum Calcium-Transporting ATPases / Q000096:biosynthesis / Q000235:genetics*
chemical_list: D009124:Muscle Proteins; D053498:Sarcoplasmic Reticulum Calcium-Transporting ATPases