Cell Manipulation Tools

项目摘要/摘要-单元格操作工具核心(工具) 人类基因组与强大的新序列导向基因相结合的最新研究进展 操纵技术彻底改变了我们提出生物问题、识别和操纵的能力 疾病引起的改变，并创造有针对性的治疗方法。然而，仍然存在严重的障碍， 试图执行和评估基因组操作的个人研究人员。这一点对于 涉及人类造血细胞产品的应用，需要专业知识和功能 化验。Tools Core的使命是充当尖端基因组操作之间的接口 技术以及研究人员和临床医生，以提供对基因治疗方案、试剂和咨询的访问 人和犬的造血细胞产品的变化。工具核心的最终目标是打破 降低访问障碍，并为功能性基因和病毒载体提供适当的基础设施和支持 造血细胞产品中的技术平台。 工具核心将由Kiem博士和Paddison博士指导，他们在以下领域拥有杰出的跟踪记录 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 祖细胞操控。 工具核心的功能包括为用户执行的基因组操作提供试剂，BEST 实践方案，造血细胞产品的操作咨询，基因编辑的细胞产品， 以及改进基因编辑和病毒载体性能的新技术的开发。

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.