Dedieu J F, Mahfoudi A, Le Roux A, Branellec D
Vector Development Department, Rhône-Poulenc Rorer Gencell, 13, quai Jules Guesde, 94403 Vitry-sur-Seine, France.
Curr Cardiol Rep. 2000 Jan;2(1):39-47. doi: 10.1007/s11886-000-0024-3.
Several phase I/II clinical trials are currently ongoing in gene therapy of cardiovascular disease. Whereas the indications vary, including peripheral artery disease, ischemic heart disease, post-angioplasty restenosis, and vein graft failure, these trials are mostly based on the use of adenoviral vectors and nonviral vectors. Novel vectors aimed at improving the efficacy and safety of gene delivery in target organs, such as heart, skeletal muscle, vasculature, and liver, have been recently generated. Some of them have already been successfully validated in preclinical models of cardiovascular disease. This review focuses on the most recent advances in vector development that could substantially increase the spectrum of cardiovascular pathologies amenable to gene transfer-based treatments.
目前有几项心血管疾病基因治疗的I/II期临床试验正在进行。尽管适应症各不相同，包括外周动脉疾病、缺血性心脏病、血管成形术后再狭窄和静脉移植物功能衰竭，但这些试验大多基于腺病毒载体和非病毒载体的使用。最近已经开发出了旨在提高基因在心脏、骨骼肌、血管系统和肝脏等靶器官中递送的疗效和安全性的新型载体。其中一些已经在心血管疾病的临床前模型中成功得到验证。本综述重点关注载体开发的最新进展，这些进展可能会大幅增加适合基于基因转移治疗的心血管疾病谱。
