GENE THERAPY IN ANIMAL MODEL OF AUTOIMMUNE DISEASE

描述：（改编自申请人摘要）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 自身免疫动物模型中的T细胞基因治疗研究方法 疾病的局限性在于不能有效地抑制鼠T细胞 细胞在过去的一年半里，Fathman实验室的工作取得了以下成果： 抗原反应性鼠T细胞的有效逆转录病毒转导。 此外，转导的T细胞的表征提供了关键的 开始在自身免疫性疾病动物模型中进行基因治疗研究所需认识 疾病这项提案将使用抗原特异性逆转录病毒修饰的T细胞 将抗炎蛋白局部递送到组织部位 炎症这些研究的结果将提供对疾病的深入了解 发病机制，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。的 这项工作的结果将对未来的 基因治疗人类疾病的结果是异常免疫 反应，即自身免疫性疾病和过敏性疾病。 主要研究者克莉丝汀博士接受过儿科培训 过敏/免疫学，并致力于学术医学事业。先前 研究经验为继续沿着这条道路前进提供了基础 Seroogy博士加入了斯坦福大学的Fathman实验室 1.5年前 从那时起，她已经获得了新的技能，在细胞 免疫学和基因治疗。额外培训年数，由 MCSDA，将允许她发展成为一个独立的调查员。

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.