Gene Transfer to Progenitor and Differentiated Cells

课程(由申请人提供)： 该计划项目的统一主题是将基因转移到与心、肺和血液疾病相关的祖细胞和分化细胞的挑战。对于这种相互竞争的更新，焦点进一步集中在与血管相关的基因转移上，特别是内皮细胞。在过去的几年里，首席研究人员发展了一系列以使用威尔·康奈尔·贝尔弗基因治疗核心设施(BGTCF)为中心的合作关系，该设施提供了12,000英尺-2的最先进的实验室空间，为基因转移载体的设计、创建、生产和评估提供专业知识和基础设施。在项目主任R.Crystal的指导下，该提案包括四个项目和四个核心，包括：项目6，S.Raffi，PI-腺病毒E4基因对血管生成的调控；项目7，B.Hempstead，PI-神经营养因子在血管生成中的作用；项目8，P.Leopold，PI-内皮腺病毒载体的转运；以及项目9，R.Crystal，PI-血管生成基因治疗中的VEGF剪接操作；Core 9001-载体；Core 9003-细胞生物学；Core 9005-分析；Core9006-实验动物。通过创造一个互动的、相互依赖的PI和协同PI的环境，具有广泛的合作历史，以及在与血管系统相关的祖细胞和/或分化细胞的生物学以及与这些细胞靶标相关的基因转移载体方面的长期经验，并利用威尔·康奈尔大学广泛的与基因转移相关的核心基础设施，已经制定了一项竞争更新，远远超出了单个研究人员或实验室能够专注于这一主题的范围。

PROGRAM (provided by applicant): The unifying theme of the Program Project is the challenge of transferring genes to progenitor and differentiated cells relevant to disorders of the heart, lung, and blood. For this competing renewal, the focus has been further concentrated on gene transfer relevant to blood vessels, particularly endothelial cells. Over the past several years, the Principal Investigators have developed a number of collaborative relationships centered on the use of the Weill Cornell Belfer Gene Therapy Core Facility (BGTCF), that provides 12,000 ft-2 of state-of-the-art laboratory space with the expertise and infrastructure for the design, creation, production, and assessment of gene transfer vectors. Under the direction of R. Crystal, Program Project Director, the proposal includes four projects and four cores, including: Project 6, S. Raffi, PI -regulation of hemangiogenesis by adenoviral E4 genes; Project 7, B. Hempstead, PI - role of the neurotrophins in angiogenesis; Project 8, P. Leopold, PI - trafficking of adenovirus vectors in endothelial cells; and Project 9, R. Crystal, PI - manipulation of VEGF splicing in angiogenic gene therapy; Core 9001-vector; Core 9003-cell biology; Core 9005-analysis; Core9006-experimental animals. By creating an interactive, interdependent environment of Pis and co-PIs with a history of extensive collaborations, and long experience in the biology of progenitor and/or differentiated cells relevant to the vascular system and gene transfer vectors relevant to these cell target, and capitalizing on the extensive gene transfer-relevant core infrastructure at Weill Cornell, a competing renewal has been crafted that far exceeds the scope of what a single investigator or laboratory could focus on this subject.