Gene and Cellular Therapy Core

基因和细胞治疗核心

• 批准号: 8631026
• 负责人: Dong Sung An
• 金额: $ 11.14万
• 依托单位: UNIVERSITY OF CALIFORNIA LOS ANGELES
• 依托单位国家: 美国
• 财政年份: 2014
• 资助国家: 美国
• 起止时间: 2014-03-01 至 2018-02-28
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/8631026
• 关键词: AIDS/HIV problem; Acquired Immunodeficiency Syndrome; Area; Basic Science; CD34 gene; Cell Separation; Cells; Collaborations; Consultations; Development; Dose; Embryo; Equipment; Gene Delivery; Genes; Genetic; Genetic Engineering; Goals; Growth; HIV; HIV Infections; HIV therapy; Hematopoietic; Human; Individual; Instruction; International; Investigation; Lentivirus Vector; Mediating; Modification; Phase I Clinical Trials; Play; Research; Research Activity; Research Personnel; Research Project Grants; Resources; Role; Services; Site; Source; Staging; Stem cells; System; T-Lymphocyte; Technical Expertise; Technology; Therapeutic; Therapeutic Studies; Training; Training Support; Transduction Gene; Translational Research; Translations; Viral Vector; Work; Zoran; base; carvedilol; cost effective; embryonic stem cell; experience; gene therapy; human embryonic stem cell; human fetus tissue; human stem cells; induced pluripotent stem cell; meetings; new technology; novel; population based; pre-clinical; research study; self-renewal; stem; stem cell technology; stem cell therapy; vector

PROJECT SUMMARY (See instructions): The overall goal of the Gene and Cellular Therapy Core (CoreG) is to provide support for HIV/AIDS-related research requiring stem cells, gene delivery vectors and technical expertise for efficient genetic modification of stem cells. Recent advancements in gene delivery vector systems and stem cell technologies have enabled genetic modification of human hematopoietic stem/progenitor cells (HSPC) to resist HIV infection. The Gene and Cellular Therapy Core is established to meet the increasing demand to promote and facilitate basic and translational research in this area by providing UCLA CFAR investigators and their domestic and international collaborators with highly purified and well characterized human CD34+ HSPC, embryonic stem cells (hESC), induced pluripotent stem cells (iPSC), human fetal tissues and lentiviral vector technologies that enable efficient genetic engineering of stem cells to resist HIV infection. The Core also provides consultation for researchers with limited experience in stem cell and viral vector technologies, in particular early stage investigators. As the use of stem cells and vector technology requires specialized expertise and resources for efficient genetic engineering of different types of stem cells, offering access to these technologies can significantly facilitate and expand the scope of UCLA CFAR research activities. Our services are more cost-effective than utilizing the limited commercial sources. Further value is added by customized technical support available from accessible and knowledgeable core staffs who can work closely with investigators to troubleshoot and optimize experiments, assist with institutional regulatory compliance documents and who are actively engaged in development and application of stem cell and vector technologies. These core services will facilitate translation of stem cell and gene therapy-related HIV research into therapeutic applications.

项目摘要(见说明)：基因和细胞治疗核心(COREG)的总体目标是为艾滋病毒/艾滋病相关研究提供支持，这些研究需要干细胞、基因传递载体和技术专长来有效地对干细胞进行基因改造。基因传递载体系统和干细胞技术的最新进展使人类造血干细胞/祖细胞(HSPC)的基因改造成为可能，以抵抗HIV感染。基因和细胞治疗核心是为了满足日益增长的需求，以促进和促进这一领域的基础和翻译研究，通过为加州大学洛杉矶分校的CFAR研究人员及其国内外合作者提供高纯度和特性良好的人CD34+HSPC、胚胎干细胞(HESC)、诱导多能干细胞(IPSC)、人类胎儿组织和慢病毒载体技术，使干细胞能够有效地进行基因工程来抵抗HIV感染。该中心还为在干细胞和病毒载体技术方面经验有限的研究人员，特别是早期研究人员提供咨询。由于干细胞和载体技术的使用需要专门知识和资源来对不同类型的干细胞进行有效的基因工程，因此提供这些技术可以极大地促进和扩大加州大学洛杉矶分校CFAR研究活动的范围。我们的服务比利用有限的商业资源更具成本效益。来自可接近和知识渊博的核心员工的定制技术支持进一步增加了价值，这些核心员工可以与调查人员密切合作，对实验进行故障排除和优化，协助机构监管合规文件，并积极参与干细胞和载体技术的开发和应用。这些核心服务将促进将与干细胞和基因治疗相关的艾滋病毒研究转化为治疗应用。