GENE THERAPY FOR EMPHYSEMA

这项研究的目的是开发一种非病毒基因 α-1-抗胰蛋白酶治疗肺气肿的给药系统 (At)缺乏的病人。AT缺乏导致蛋白水解酶诱导 肺损伤，可发展为肺气肿。目前在那里 没有足够的手段来抑制疾病的发展。AT蛋白质 治疗已被证明只能暂时改善疾病。 病毒基因递送系统正在开发中的进展 这种方法将受到免疫学的局限性的阻碍。 宿主的反应。非病毒基因疗法有可能 绕过许多免疫学和传染性问题 与病毒基因治疗相关，并提供更持久的 对肺气肿患者的治疗优于蛋白质治疗。我们假设 阳离子脂质体基因传递系统可以有效地 肺上皮细胞。改善核酸在肺内的转运 组织将允许足够的AT蛋白产生到 足以对抗肺泡破坏并逆转或防止 疾病。这项研究将提供对参数的洞察 管理阳离子脂质体介导的基因传递，并计算 方法和计算机图形将允许更合理的设计 用于基因治疗的后续几代阳离子脂质体。

The purpose of this research is to develop a non-viral gene delivery systems for the treatment of emphysema in alpha-1-antitrypsin (AT) deficient patients. AT deficiency leads to protease-induced damage in the lung which can develop into emphysema. Currently there are no adequate means to inhibit disease progression. AT protein therapy has been shown to only temporarily ameliorate disease progression while viral gene delivery systems are under development this approach will be hampered by limitations of immunological reaction of the host. Non-viral gene therapy has the potential to circumvent many of the immunological and infectivity problems associated with viral gene therapy and provide for a more durable treatment than protein therapy for emphysema patients. We hypothesize that cationic liposome gene delivery systems can efficiently transfect lung epithelial cells. Improvement of nucleic acid delivery to lung tissue will allow adequate production of AT protein to levels sufficient to combat alveolar destruction and reverse or prevent disease. This research will provide insight into the parameters governing cationic liposome-mediated gene delivery, and computational approaches and computer graphics will permit a more rational design of subsequent generations of cationic liposomes for gene therapy.