新型聚乙烯亚胺复合干扰表达血管生长因子基因治疗婴幼儿血管瘤的作用及机制研究

Abstract: Hemangioma is the most common congenital benign tumor among infants, which proliferates rapidly within one year and then enter the slow process of self-involuting. Multiple or severe hemangiomas in the proliferative phase can cause serious complications, which may affect the physiological function and even endanger the lives. Thus, early and aggressive intervention must be done. There is no completely effective treatment method for hemangiomas because of its unclear pathogenesis. Our previous study found that delivery of anti-angiogenic growth factor gene (RNAi) by synthetic delivery vehicle of efficiency and low toxicity can inhibit neovascularization of hemangiomas. On this basis, this study will further explore how the delivery of anti-angiogenic growth factor gene is able to treat hemangiomas. We study the inhibitory effect of delivery of anti-angiogenic growth factor gene for hemangioma endothelial cells and hemangioma stem cells and its mechanism in vitro and in vivo with hemangioma nude model as a platform. Our aim is to convey a clear treatment effect of delivering anti-angiogenic growth factor gene and investigate the mechanism. This can lay the foundation of delivering anti-angiogenic growth factor gene to treat hemangiomas.

血管瘤是婴幼儿最常见的先天性良性肿瘤，1岁内快速增殖，随后进入缓慢的自行消退过程。增殖期的多发性或重症血管瘤可引起严重并发症，影响生理功能甚至危及生命，必须早期积极干预。由于其发病机制不清楚，目前尚无一种完全有效的治疗方法。课题组前期研究发现，利用合成高效低毒输送载体输送干扰（沉默）表达血管生长因子基因（RNAi），能够抑制血管瘤的新生血管形成，在此基础上，本研究将进一步探索输送干扰（沉默）表达血管生长基因对血管瘤的治疗作用，以血管瘤裸鼠模型为平台，从体内和体外研究输送干扰（沉默）表达血管生长基因对血管瘤内皮细胞和血管瘤干细胞的抑制作用及其机制，明确输送干扰（沉默）表达血管生长因子基因的治疗效果，并探讨其作用机制，为输送干扰（沉默）表达血管生长因子基因治疗血管瘤奠定基础。

血管瘤是婴幼儿最常见的先天性良性肿瘤，1岁内快速增殖，随后进入缓慢的自行消退过程。增殖期的多发性或重症血管瘤可引起严重并发症，影响生理功能甚至危及生命，必须早期积极干预。由于其发病机制不清楚，目前尚无一种完全有效的治疗方法。 我们合成了一系列的高效低毒输送载体材料输送干扰表达血管生长因子基因（RNAi），能够抑制血管瘤的新生血管形成，并进一步验证输送干扰（沉默）表达血管生长基因对血管瘤的治疗作用，以血管瘤裸鼠模型为平台，从体内和体外研究和证实了输送干扰（沉默）表达血管生长基因对血管瘤的抑制作用及其相关机制，明确输送干扰（沉默）表达血管生长因子基因的治疗效果，为输送干扰（沉默）表达血管生长因子基因治疗血管瘤奠定基础。并开发出一种用于凝胶透皮治疗婴幼儿血管瘤的新药，并获得授权发明专利，转让到相关的医药公司进一步开发。同时还发现一种高效建立婴幼儿血管瘤的模型方法和扩充了输送干扰表达血管生长因子基因（RNAi）或促进血管形成基因在别的肿瘤或其它疾病应用研究，也取得一定的疗效。

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