期刊
EUROPEAN HEART JOURNAL
卷 37, 期 21, 页码 1651-1658出版社
OXFORD UNIV PRESS
DOI: 10.1093/eurheartj/ehw019
关键词
Gene therapy; Heart failure; Adeno-associated vectors; Sarcoplasmic reticulum calcium ATPase; Excitation-contraction coupling
资金
- National Institutes of Health [R01 HL117505, HL119046, HL128072, HL129814, P50 HL112324, T32HL007824, R01 HL113497]
- Transatlantic Fondation Leducq grant
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.
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