Core D: Genetic Engineering and Gene Therapy Core

基因工程和基因治疗(GEGT)的核心任务是与肝脏合作 研究中心(LRC)研究人员为开发用于In的基因工程试剂提供便利 体外和体内实验。其具体目标是(1)产生定制的肿瘤逆转录病毒、慢病毒、 具有整合或附体特征的腺病毒和非病毒载体；(2)提供最先进的 用于细胞标记、表型校正/修饰或下调的位点特异性基因插入试剂 基因表达；(3)提供病毒和非病毒载体，用于重新编程体细胞以 诱导多能干细胞(IPS)；(4)适当时，培训研究人员及其研究人员 在自己的实验室生成基因转移载体；(5)为研究设计提供咨询，如果 适当地，对调查人员进行数据分析。为了满足这些需求，GEGT通过一个 与阿尔伯特·爱因斯坦学院基因治疗核心的合作费用分担安排 医学。GEGT核心将与其他LRC核心以及其他核心密切合作 机构共享设施，但GEGT提供的专门服务与 其他核心。核心将受益于科学总监Jayanta Roy长达25年的经验- 乔杜里教授，医学和遗传学教授，研究体内和体外的致病生物学和基因转移 翻译研究，以及运营总监夏博士的重要技术专长 王。GEGT Core将为LRC调查人员增加价值，使他们能够生成和使用矢量和 对于个别实验室来说，开发这种试剂的成本高得令人望而却步，或者在技术上存在困难。它 还将为LRC调查人员提供优先服务和折扣的退款费率。资源共享与 其他LRC核心以及其他机构范围的共享设施将允许核心提供其服务 以有效和具有成本效益的方式。通过支持多方面的病理生理和翻译 法改会成员的研究项目，以及校外调查人员，核心将高度相关 与医疗保健相关的肝脏研究。

The mission ofthe Genetic Engineering and Gene Therapy (GEGT) Core is to work cooperatively with Liver Research Center (LRC) Investigators to facilitate the development of genetic engineering reagents for in vitro and in vivo experiments. The specific aims are to (1) generate customized oncoretroviral, lentiviral, adenoviral and non-viral vectors with integrating or episomal characteristics; (2) provide state-of-the-art reagents for site-specific gene insertion for cell marking, phenotypic correction/modification or down-regulating gene expression; (3) provide viral and non-viral vectors for reprogramming somatic cells to induced pluripotent stem (IPS) cells; (4) when appropriate, train investigators and their research personnel in generating gene transfer vectors in their own laboratories; (5) provide consultation for study design and, if appropriate, data analysis to the investigators. To fulfill these needs, the GEGT is established through a cooperative cost-sharing arrangement with the Gene Therapy Core of the Albert Einstein College of Medicine. The GEGT Core will function in close collaboration with other LRC Cores, as well as other institutional Shared Facilities, but the specialized services of the GEGT do not overlap with those offered by other Cores. The Core will benefit from the 25-yr experience of the Scientific Director, Jayanta Roy- Chowdhury, Professor of Medicine and Genetics in gene transfer in vivo and ex vivo for pathobiological and translational research, as well as the significant technological expertise of the Operations Director, Dr. Xia Wang. GEGT Core will add value to LRC investigators by enabling them to generate and use vectors and reagents that would be prohibitively expensive or technically difficult for individual laboratories to develop. It will also give LRC investigators priority for service and discounted chargeback rates. Resource sharing with other LRC Cores, as well as other institution-wide shared facilities will allow the Core to provide its services in an effective and cost-efficient manner. By supporting the multifaceted pathophysiological and translational research projects of the LRC members, as well as extramural investigators, the Core will be highly relevant to healthcare-related liver research.