Center for Gene Therapy of Cystic Firbosis and Other Genetic Diseases

囊性纤维化和其他遗传疾病基因治疗中心

• 批准号: 7835846
• 负责人: JOHN F ENGELHARDT
• 金额: $ 112.5万
• 依托单位: UNIVERSITY OF IOWA
• 依托单位国家: 美国
• 财政年份: 1998
• 资助国家: 美国
• 起止时间: 1998-09-30 至 2014-03-31
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/7835846
• 关键词: Center; Gene; Therapy; Cystic; Firbosis

DESCRIPTION, OVERALL (provided by applicant): The development of effective gene therapies for inherited disorders requires interdisciplinary programs that focus on the identification of disease genes, an elucidation of disease pathogenesis, and the development of technologies for genetic modulation. The Center's goal has been to provide investigators with the opportunity to improve and/or expand their gene therapy-based research. Although this Center has a primary focus on the development of gene therapies for cystic fibrosis (CF), as evidenced through two of its Research Cores that apply specialized airway model systems to CF research, it also maintains active research programs in molecular medicine for a diverse range of other genetic diseases. The Center has productively fostered gene therapy research through the following mechanisms. 1) The Center's Pilot and Feasibility Program, which has sponsored 50 pilots over the previous 9 years of funding, has brought numerous new Members and expertise into the Center while fostering the development of talented junior Associate Members into independent tenure-track faculty positions. 2) The Center has successfully established and expanded existing specialized Core facilities (Vector Core, Cell and Tissue Core, Cell Morphology and Pathology Core, and Animal Models Core) that are devoted to gene therapy research. These Cores have provided Center investigators with specialized vectors, human and mouse CF model systems, and methods that have allowed them to test hypotheses that could not otherwise be evaluated. The Center has also served as a resource for the distribution of viral vectors and CF models to numerous outside institutions. 3) By combining viral gene-targeting technologies with animal modeling, the Center has also created two new larger CF animal models, in the pig and ferret, to address the growing need for phenotypic models of human CF lung disease. 4) The Center has successfully maintained a CF Lung Tissue Acquisition Consortium of lung transplant centers, which has allowed for significant expansion of the humanized CF model systems. This consortium, which receives between 15-20 CF lung transplants per year, has also promoted numerous intrainstitutional collaborations on CF research that have led to new research programs. 5) The Center has also fostered interdisciplinary interactions and training through its Enrichment Programs, consisting of a centralized center website, a trainee seminar series, an external speaker seminar series, smaller informal research focus groups, and the annual Gene Therapy Center Retreat. 6) The Center has established formal internal and external mechanisms for review of the Center, the Cores, and the Pilot and Feasibility projects, thereby ensuring a high level of excellence and the most appropriate utilization of the Center's resources. In summary, the Center for Gene Therapy of Cystic Fibrosis and Other Genetic Diseases has greatly strengthened existing gene therapy-based research programs at the University of Iowa and consortium institutions, while also serving as a resource for other institutions that perform gene therapy and CF research. These efforts have led to numerous basic and applied research findings that have enhanced the utility of gene therapies to both study and treat genetic diseases.

总体描述（由申请人提供）：遗传性疾病的有效基因疗法的开发需要跨学科项目，这些项目侧重于疾病基因的鉴定、疾病发病机制的阐明以及遗传调节技术的开发。该中心的目标是为研究人员提供改善和/或扩大其基因治疗研究的机会。尽管该中心的主要重点是囊性纤维化（CF）基因疗法的开发，正如其两个将专门的气道模型系统应用于CF研究的研究核心所证明的那样，但它还在分子医学方面保持着积极的研究计划，用于多种疾病。其他遗传疾病。该中心通过以下机制卓有成效地促进了基因治疗研究。1)该中心的试点和可行性计划，在过去9年的资助中赞助了50个试点项目，为中心带来了许多新成员和专业知识，同时促进了有才华的初级准成员发展成为独立的终身教职。2)该中心已成功地建立和扩大了现有的专门核心设施（载体核心，细胞和组织核心，细胞形态学和病理学核心和动物模型核心），致力于基因治疗研究。这些核心为中心研究人员提供了专门的载体，人类和小鼠CF模型系统，以及允许他们测试假设的方法，否则无法进行评估。该中心还作为向许多外部机构分发病毒载体和CF模型的资源。3)通过将病毒基因靶向技术与动物建模相结合，该中心还在猪和雪貂中创建了两种新的较大的CF动物模型，以满足对人类CF肺病表型模型日益增长的需求。4)该中心成功地维持了肺移植中心的CF肺组织采集联盟，这使得人源化CF模型系统得到了显著扩展。该联盟每年接受15-20例CF肺移植手术，还促进了CF研究方面的许多机构内合作，这些合作导致了新的研究项目。5)该中心还通过其丰富计划促进了跨学科的互动和培训，包括一个集中的中心网站，一个实习生研讨会系列，一个外部演讲者研讨会系列，较小的非正式研究重点小组，以及年度基因治疗中心务虚会。6)中心建立了正式的内部和外部机制，对中心、核心项目、试点项目和可行性项目进行审查，从而确保中心的资源得到高度的优化和最适当的利用。总之，囊性纤维化和其他遗传性疾病基因治疗中心大大加强了爱荷华州大学和联盟机构现有的基于基因治疗的研究计划，同时也为其他进行基因治疗和CF研究的机构提供了资源。这些努力导致了许多基础和应用研究的发现，这些发现增强了基因疗法在研究和治疗遗传疾病方面的效用。