Establishment of Molecular Therapy for Severe Heart Failure and Intractable Fetal Arrhythmia

靶向钙循环已成为治疗严重心力衰竭的一种潜在疗法。在这里，我们开发了一种新型的心力衰竭诱导的基因转移系统，通过腺相关病毒载体9(AAV9)介导的shRNA抑制PP1。AAV9介导的基因转移减轻了肌肉LIM蛋白缺陷(MLPKO)小鼠心肌病的进行性左室重构和改善心力衰竭进展。心力衰竭诱导基因治疗系统为治疗严重心力衰竭和难治性胎儿心律失常提供了新的潜力。

The targeting of Ca^<2+> cycling has emerged as a potential therapy for the treatment of severe heart failure. Here, we developed a novel heart-failure-inducible gene transfer system that inhibits PP1 by adeno-associated virus vector 9(AAV9)-mediated shRNA. AAV9 mediated gene transfer mitigated progressive left ventricular remodeling and ameliorated heart failure progression in muscle LIM proteindeficient(MLPKO) mice cardiomyopathy. Heart failure inducible gene therapy system provides new potential as a strategy of treatment of severe heart failure and intractable fetal arrhythmia.