Sarcoplasmic reticulum and calcium cycling targeting by gene therapy

• Published in: Gene therapy Vol. 19; no. 6; pp. 596 - 599
• Main Authors: Hulot, J-S, Senyei, G, Hajjar, R J
• Format: Journal Article
• Language: English
• Published: England Nature Publishing Group 01.06.2012
• Subjects: Animals; Ca super(2+)-transporting ATPase; Ca2+-transporting ATPase; Calcium; Calcium (reticular); Calcium ions; Cardiovascular disease; Care and treatment; Clinical Trials as Topic; Congestive heart failure; Coronary artery; Diabetes mellitus; Etiology; Evolution; Gene Targeting - methods; Gene therapy; Gene Transfer Techniques; Genetic aspects; Genetic Therapy - methods; Health aspects; Heart diseases; Heart failure; Heart Failure - therapy; Humans; Hypertension; Infection; Inflammation; Mortality; Myocytes; Physiological aspects; Sarcoplasmic reticulum; Sarcoplasmic Reticulum Calcium-Transporting ATPases - genetics; United States
• ISSN: 0969-7128; 1476-5462
• DOI: 10.1038/gt.2012.34

Although progress in conventional treatments is making steady and incremental gains to reduce mortality associated with heart failure (HF), there remains a need to explore potentially new therapeutic approaches. HF induced by different etiologies such as coronary artery disease, hypertension, diabetes, infection or inflammation results generally in calcium cycling dysregulation at the myocyte level. Recent advances in understanding of the molecular basis of these calcium cycling abnormalities, together with the evolution of increasingly efficient gene transfer technology, has placed HF within the reach of gene-based therapy. Furthermore, the recent successful completion of a phase 2 trial targeting the sarcoplasmic reticulum calcium pump ushers in a new era for gene therapy for the treatment of HF.