Gene Delivery Core

摘要 血液病的分子和细胞病理生理学的研究需要快速， 以低成本获得病媒制剂，以及获得大型病媒库和生产病媒 用于高通量筛选。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”， 在基础研究、研究载体和临床载体制造实验室之间建立联系。辛辛那提 儿童医院医疗中心是一家卓越的临床和研究机构， 为血液学研究者提供生物学研究的载体支持， 细胞的遗传操作及其临床应用。这个核心有一个跟踪记录， 为基础研究各阶段的研究人员提供高质量的“研究级”和“临床规模”病媒服务 和翻译研究，确保迅速翻译到诊所。基因的组成部分 有助于这一努力的交付核心（GDC）是病毒载体核心和载体库核心。每个 这些子核心都配备了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，其在常规基础研究实验室研究中的评价和/或 高通量筛选，以及随着研究通过转化 “管道”。所有这些努力都得到了机构对转化研究的坚定承诺的支持， 广泛的血液学领域用户的普遍需求。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）载体库核心服务，包括生产慢病毒shRNA和CRISPR 用于高通量筛选的gRNA文库。我们的总体目标是推进研究和开发， 通过促进基因递送、基因敲除、基因敲除或基因编辑， 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.