Cell Manipulation Tools

项目总结/摘要-细胞操作工具核心（工具） 人类基因组的最新特征与强大的新序列指导基因相结合 操纵技术已经彻底改变了我们提出生物学问题的能力， 疾病引起的改变，并创造有针对性的治疗。然而，仍然存在严重的障碍， 个体研究人员试图执行和评估基因组操作。尤其如此 涉及人类造血细胞产品的应用，需要专业知识和功能 测定。Tools Core的使命是充当尖端基因组操作 技术和研究人员和临床医生提供访问协议，试剂和遗传咨询， 人类和犬造血细胞产物的改变。工具核心的最终目标是打破 降低获取障碍，为功能性遗传和病毒载体提供适当的基础设施和支持 造血细胞产品的技术平台。 工具核心将由Kiem博士和Paddison博士指导，他们在以下领域拥有出色的记录： 病毒载体介导的基因治疗，功能基因组学，基因编辑，载体生产，以及干细胞和 祖细胞操作 工具核心的功能包括为用户执行的基因组操作提供试剂， 操作造血细胞产品、基因编辑的细胞产品的实践方案、咨询， 以及开发新技术以改善基因编辑和病毒载体性能。

PROJECT SUMMARY/ABSTRACT - Cell Manipulation Tools Core (Tools) The recent characterization of the human genome combined with powerful new sequence-directed gene manipulation technologies have revolutionized our ability to ask biological questions, identify and manipulate disease causing alterations, and create targeted therapeutics. However, serious barriers still exist for individual researchers attempting to perform and assess genome manipulations. This is particularly true for applications involving human hematopoietic cell products, which require specialized knowledge and functional assays. The mission of the Tools Core is to act as an interface between cutting edge genome manipulation techniques and researchers and clinicians to provide access to protocols, reagents, and consultants for genetic alteration of human and canine hematopoietic cell products. The ultimate goal of the Tools Core is to break down access barriers and provide appropriate infrastructure and support for functional genetic and viral vector technology platforms in hematopoietic cell products. The Tools Core will be directed by Drs. Kiem and Paddison who have outstanding track records in the fields of viral vector-mediated gene therapy, functional genomics, gene editing, vector production, and stem and progenitor cell manipulation. Functions of the Tools Core include provision of reagents for user-performed genome manipulations, best practices protocols, consultations for manipulation of hematopoietic cell products, gene edited cell products, and development of new technology to improve gene editing and viral vector performance.