GENE THERAPY IN ANIMAL MODEL OF AUTOIMMUNE DISEASE

描述：(改编自申请人的摘要)基因的应用 自身免疫性疾病动物模型的治疗将解决相关问题 自身免疫性疾病和免疫反应的改变。有意义的 自身免疫动物模型中T细胞基因治疗的研究进展 疾病一直受到无法有效地转导小鼠T细胞的限制 细胞。在Fathman实验室过去1.5年的工作中， 抗原反应性小鼠T细胞的有效逆转录病毒转导。 此外，转导的T细胞的特征提供了关键的 在自身免疫动物模型中开始基因治疗研究需要洞察力 疾病。这项提议将使用抗原特异性逆转录病毒修饰的T细胞 将抗炎蛋白局部输送到组织部位 发炎。这些研究的结果将提供对疾病的洞察 致病机制，局部递送疾病修饰蛋白的能力 自身抗原反应性T细胞，以及改变免疫反应的能力。这个 这项工作的发现将对未来的 异常免疫导致的人类疾病的基因治疗 反应，即自身免疫性疾病和过敏性疾病。 首席调查员克里斯汀·塞洛格博士接受过儿科培训 过敏症/免疫学，致力于学术医学事业。上一首 研究经验为继续沿着这条道路前进提供了基础 职业道路。Seroology博士加入了斯坦福大学的Fathman实验室 一年半以前。从那时起，她获得了细胞方面的新技能 免疫学和基因疗法。额外的几年培训，由 MCSDA，将允许她发展成为一名独立的调查员。

DESCRIPTION: (adapted from applicant's abstract) The application of gene therapy in animal models of autoimmune disease will address questions relevant to autoimmune diseases and the alteration of immune responses. Meaningful T-cell gene therapy investigative approaches in animal models of autoimmune disease have been limited by the inability to efficiently transduce murine T cells. Work over the past 1.5 years in the Fathman laboratory has resulted in efficient retroviral transduction of antigen-reactive murine T cells. Furthermore, characterization of the transduced T cells has provided critical insight needed to begin gene therapy studies in animal models of autoimmune disease. This proposal will use antigen-specific retrovirally-modified T cells to locally deliver anti-inflammatory proteins to the site of tissue inflammation. The results from these studies will provide insight into disease pathogenesis, the ability to locally deliver disease-modifying proteins with autoantigen-reactive T cells, and the ability to alter immune responses. The findings from this work will have important implications for the future of gene therapy in human diseases that are the result of aberrant immune responses, i.e. autoimmune diseases and allergic diseases. The Principal Investigator, Dr. Christine Seroogy, is trained in pediatric allergy/immunology and is committed to a career in academic medicine. Previous research experience has provided the foundation for continuing along this career path. Dr. Seroogy joined the Fathman laboratory at Stanford University 1.5 years ago. Since that time she has acquired new skills in cellular immunology and gene therapy. Additional years of training, provided by the MCSDA, will allow her to develop into an independent investigator.