Gene Delivery Core

摘要 血液病的分子和细胞病理生理学研究需要快速和 以低成本获得病媒准备，以及获得大型病媒文库和生产病媒 注定要进行高通量筛选。此外，新基因的成功开发和测试 治疗需要进入翻译载体“管道”，这就需要密切的互动 基础研究、研究载体和临床媒介制造实验室之间的联系。辛辛那提 儿童医院医学中心，一家卓越的临床和研究机构 杰出的核心为血液学研究人员提供载体支持生物研究 细胞的遗传操作及其在临床上的应用。这个核心有提供以下服务的记录 为基础设施不同阶段的研究人员提供高质量的“研究级”和“临床规模”的媒介服务 和翻译研究，确保快速翻译到临床。基因的组成成分 促成这一努力的交付核心(GDC)是病毒载体核心和矢量库核心。每个人 这些子核心中有自己的最先进的研究实验室，以允许快速访问 载体文库和病毒载体产品，它们在常规基础研究实验室研究和/或 高通量筛查，以及随着研究的进行将其翻译到临床 “管道”。所有这些努力都得到了机构对翻译研究和 广泛流行于广大血液学领域的用户需求。我们的目标是：(1) 研究规模和中等规模的中试和可行性病毒载体产品和载体类型咨询 和设计，以及(2)载体文库核心服务，包括生产慢病毒shRNA和CRISPR 高通量筛选的gRNA文库。我们的总体目标是在 血液学通过促进基因传递、基因敲除、基因敲除或基因编辑来加速 翻译的速度。

Abstract Investigation of the molecular and cellular pathophysiology for hematologic diseases requires rapid and low-cost access to vector preparations, and access to large libraries of vectors and production of vectors destined to high throughput screening. Further, the successful development and testing of novel gene therapies requires access to a translational vector “pipeline” which necessitates a close interaction between basic research, research vector and clinical vector manufacturing laboratories. Cincinnati Children’s Hospital Medical Center, a clinical and research institution of excellence, has developed outstanding cores to provide hematology investigators with vector support for biological studies for the genetic manipulation of cells and their translation to the clinic. This core has a track record of providing high quality “research-grade” and “clinical-scale” vector services to investigators at various stages of basic and translational research, ensuring expeditious translation to the clinic. The components of the Gene Delivery Core (GDC) that contribute to this effort are the Viral Vector Core and Vector Library Core. Each of these sub-cores are equipped with its own state-of-the-art research laboratories to allow rapid access to vector libraries and viral vector products, their evaluation in routine basic research lab studies and/or high throughput screens, and their translation to the clinic as the studies move through the translational “pipeline”. All of this effort is backed by a strong Institutional commitment to translational research and widely popular demands from users in the broad hematology field. Our objectives are to provide: (1) research scale and medium scale pilot and feasibility viral vector products and consultation on vector type and design, and (2) vector library core services including production of lentiviral shRNA and CRISPR gRNA libraries for high throughput screening. Our overall goal is to advance research and development in hematology via facilitating gene delivery, gene knock down, gene knockout or gene editing, to accelerate the rate of translation.