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高效转染血管平滑肌细胞的翻译型 AAV2.5 载体:支架内再狭窄基因治疗的新视角。

Efficient transduction of vascular smooth muscle cells with a translational AAV2.5 vector: a new perspective for in-stent restenosis gene therapy.

机构信息

INSERM, UMRS 956, Paris, France.

出版信息

Gene Ther. 2013 Sep;20(9):901-12. doi: 10.1038/gt.2013.13. Epub 2013 Mar 28.

Abstract

Coronary artery disease represents the leading cause of mortality in the developed world. Percutaneous coronary intervention involving stent placement remains disadvantaged by restenosis or thrombosis. Vascular gene therapy-based methods may be approached, but lack a vascular gene delivery vector. We report a safe and efficient long-term transduction of rat carotid vessels after balloon injury intervention with a translational optimized AAV2.5 vector. Compared with other known adeno-associated virus (AAV) serotypes, AAV2.5 demonstrated the highest transduction efficiency of human coronary artery vascular smooth muscle cells (VSMCs) in vitro. Local delivery of AAV2.5-driven transgenes in injured carotid arteries resulted in transduction as soon as day 2 after surgery and persisted for at least 30 days. In contrast to adenovirus 5 vector, inflammation was not detected in AAV2.5-transduced vessels. The functional effects of AAV2.5-mediated gene transfer on neointimal thickening were assessed using the sarco/endoplasmic reticulum Ca(2+) ATPase isoform 2a (SERCA2a) human gene, known to inhibit VSMC proliferation. At 30 days, human SERCA2a messenger RNA was detected in transduced arteries. Morphometric analysis revealed a significant decrease in neointimal hyperplasia in AAV2.5-SERCA2a-transduced arteries: 28.36±11.30 (n=8) vs 77.96±24.60 (n=10) μm(2), in AAV2.5-green fluorescent protein-infected, P<0.05. In conclusion, AAV2.5 vector can be considered as a promising safe and effective vector for vascular gene therapy.

摘要

冠状动脉疾病是发达国家死亡的主要原因。涉及支架放置的经皮冠状动脉介入术仍然存在再狭窄或血栓形成的风险。可以采用基于血管基因治疗的方法,但缺乏血管基因传递载体。我们报告了一种经过优化的翻译 AAV2.5 载体,在球囊损伤干预后,对大鼠颈动脉进行了安全有效的长期转导。与其他已知的腺相关病毒(AAV)血清型相比,AAV2.5 在体外对人冠状动脉血管平滑肌细胞(VSMCs)的转导效率最高。在损伤的颈动脉中局部递送 AAV2.5 驱动的转基因,可在手术后第 2 天开始转导,并持续至少 30 天。与腺病毒 5 载体相反,在 AAV2.5 转导的血管中未检测到炎症。使用肌浆/内质网 Ca(2+)ATP 酶同工型 2a(SERCA2a)人类基因评估 AAV2.5 介导的基因转移对新生内膜增厚的功能影响,该基因已知可抑制 VSMC 增殖。在 30 天时,在转导的动脉中检测到人类 SERCA2a 信使 RNA。形态计量分析显示,AAV2.5-SERCA2a 转导动脉中的新生内膜过度增生显著减少:28.36±11.30(n=8)μm(2) vs 77.96±24.60(n=10)μm(2),AAV2.5-绿色荧光蛋白感染的,P<0.05。总之,AAV2.5 载体可以被认为是一种有前途的安全有效的血管基因治疗载体。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/d1e7/3706517/068145a2a691/nihms-442952-f0001.jpg

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