GENE THERAPY FOR EMPHYSEMA

肺气肿的基因治疗

• 批准号: 6280278
• 负责人: ROM ELIAZ
• 金额: $ 0.01万
• 依托单位: UNIVERSITY OF CALIFORNIA, SAN FRANCISCO
• 依托单位国家: 美国
• 财政年份: 1998
• 资助国家: 美国
• 起止时间: 1998-07-01 至 1999-06-30
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/6280278
• 关键词: Mammalia; animal tissue; biological products; biomedical resource; biotechnology; drug /agent; genetics; human tissue; respiratory system; technology /technique

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.

这项研究的目的是开发一种非病毒基因 用于治疗肺气肿的α-1-抗胰蛋白酶递送系统 (AT)有缺陷的病人 AT缺乏导致蛋白酶诱导的 肺损伤可能发展为肺气肿。 当前 并不足以抑制疾病的发展。 AT蛋白 治疗已被证明只能暂时改善疾病 病毒基因传递系统正在开发中 这种方法将受到免疫学的局限性的阻碍， 主持人的反应。 非病毒基因疗法有可能 避免了许多免疫和传染问题， 与病毒基因治疗相关，并提供更持久的 对于肺气肿患者来说，蛋白质治疗比蛋白质治疗更有效。 我们假设 阳离子脂质体基因传递系统可以有效地抑制 肺上皮细胞 向肺递送核酸的改进 组织将允许足够的AT蛋白的产生， 足以对抗肺泡破坏并逆转或防止 疾病 这项研究将提供深入了解参数 控制阳离子脂质体介导的基因递送， 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。