BCM-Rice resource for the analysis of somatic gene editing in mice

用于分析小鼠体细胞基因编辑的 BCM-Rice 资源

• 批准号: 10217283
• 负责人: Mary E Dickinson
• 金额: $ 68.5万
• 依托单位: BAYLOR COLLEGE OF MEDICINE
• 依托单位国家: 美国
• 财政年份: 2018
• 资助国家: 美国
• 起止时间: 2018-09-18 至 2023-07-31
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/10217283
• 关键词: Address; Animal Model; Award; Benefits and Risks; Clinical Trials; Communities; Commuting; Data; Deposition; Elements; Evaluation; Genes; Goals; Housing; Human Resources; Infrastructure; Knockout Mice; Leadership; Medical center; Modeling; Mouse Strains; Mus; Phenotype; Production; Reporter; Research; Research Personnel; Resources; Rice; Safety; Services; Site; Specificity; System; Technology; Testing; Texas; Tissues; Work; animal care; base; clinical development; collaborative environment; community center; efficacy evaluation; experience; gene therapy; genome editing; human disease; in vivo evaluation; member; mouse model; novel; programs; public repository; scientific organization; somatic cell gene editing; tool; translational approach

Project Summary – Overall Genome editing systems have the potential to cure some of the most severe human diseases. However, there are significant challenges that must be addressed before this technology can be applied in clinical trials including identification of appropriate delivery systems for specific tissues, evaluation of overall efficiency and specificity of delivery and genome editing systems, and assessment of efficacy and safety of both delivery and genome editing technologies. The BCM-Rice Resource Center for the Analysis of Somatic Gene Editing in Mice will create mouse model resources for in vivo testing of somatic genome editing tools developed by members of the Somatic Cell Genome Editing (SCGE) program, will assist researchers within the program with evaluation of genome editing strategies using these new models, and will deposit genome editing reporter mouse strains into a publically accessible repository for distribution to the wider scientific community. Our long term goal is to accelerate the clinical development of somatic genome editing for human disease by defining the benefits and risks of novel Delivery Technologies and Editing Systems. The Overall section provides a statement of significance, a synopsis of the work to be performed, relevant descriptions of the resources available for this project at BCM and Rice, a description of the qualifications of the PIs and research team and a description of previous expertise and infrastructure relevant to this proposal. Together, these sections convey the strengths of BCM-Rice as a proposed site for a new Resource Center for the Analysis of Somatic Gene Editing. It is clear that the Texas Medical Center (TMC) community will bring significant benefits to the project and this project will also strongly benefit the TMC community.

项目摘要-整体