GENE THERAPY FOR AUTOIMMUNE ARTHRITIS

自身免疫性关节炎的基因疗法

• 批准号: 6403162
• 负责人: SHERRY L THORNTON
• 金额: $ 4.38万
• 依托单位: CINCINNATI CHILDRENS HOSP MED CTR
• 依托单位国家: 美国
• 财政年份: 2001
• 资助国家: 美国
• 起止时间: 2001-01-04 至
• 项目状态: 未结题
• 来源: https://reporter.nih.gov/project-details/6403162
• 关键词: arthritis; autoimmune disorder; biotechnology; gene therapy; immune tolerance /unresponsiveness; interleukin 10; laboratory mouse; nonhuman therapy evaluation; transfection /expression vector

Gene therapy provides a possible means to deliver anti-inflammatory biological agents, such as interleukin-10, for long-term treatment of autoimmune arthritis. Adenoviral vector mediated gene therapy allows for high expression of these therapeutic proteins. However, this expression is normally short-lived, in part due to immunogenicity of the viral vector and/or transgenic protein being expressed. Studies outlined in the first two specific aims will determine whether transgene expression can be prolonged utilizing adenoviral vectors engineered to reduce immunogenicity to the adenoviral vector and/or transgenic protein product. The third specific aim functionally tests these vectors in the CIA mouse model system for their effects on autoimmune arthritis. The studies outlined in this proposal will provide valuable data in furthering the development of gene therapy for autoimmune arthritis.

基因疗法提供了一种可能提供抗炎生物学剂（例如白介素10）的方法，用于长期治疗自身免疫性关节炎。腺病毒载体介导的基因疗法可以高表达这些治疗蛋白。但是，这种表达通常是短暂的，部分是由于病毒载体和/或转基因蛋白的免疫原性。 在前两个具体目标中概述的研究将确定转基因表达是否可以延长使用腺病毒载体，以降低对腺病毒载体和/或转基因蛋白产物的免疫原性。第三个特定的目标在功能上测试了CIA小鼠模型系统中的这些向量，以便它们对自身免疫性关节炎的影响。该提案中概述的研究将提供有价值的数据，以进一步发展自身免疫性关节炎的基因疗法。