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Gene Editing Core

基因编辑核心

基本信息

批准号：
10668765
负责人：
DAVID R LIU
金额：
$ 40.85万
依托单位：
JACKSON LABORATORY
依托单位国家：
美国
项目类别：
财政年份：
2023
资助国家：
美国
起止时间：
2023-05-16 至 2028-04-30
项目状态：
未结题

来源：
https://reporter.nih.gov/project-details/10668765
关键词：
Animals Archives Biological Biological Assay Biological Models Cell Line Cell Separation Cells Clinic Complex Custom DNA DNA Double Strand Break Data Deaminase Ensure Enzymes Equipment Event Future Genes Genome Genomic DNA Genomics Goals High-Throughput Nucleotide Sequencing In Vitro Individual Laboratories Libraries Mammalian Cell Methods Monitor Outcome Play Process Program Efficiency Recording of previous events Reporting Research Project Grants Ribonucleoproteins Role Running Sampling Site Technology Therapeutic Tissue Sample Tissues Validation Viral Vector analysis pipeline animal tissue base editing candidate identification cell type data analysis pipeline design detection method efficacy study genome editing genome-wide human DNA in vivo member mouse model nervous system disorder off-target site pre-clinical prevent prime editing programs success synergism therapeutic candidate therapeutic genome editing therapeutic target tool vector

项目摘要

PROJECT SUMMARY GENE EDITING CORE The Liu laboratory at the Broad Institute of MIT and Harvard will serve as the Gene Editing Core for the Program. Developing customized editing strategies that maximize therapeutic editing while minimizing undesired byproducts is important for the success of all Research Projects. A centralized Gene Editing Core staffed by Liu laboratory members will enhance the Program's efficiency by preventing bottlenecks in gene editing analyses and ensuring consistency in methods and materials used in the proposed projects. The Gene Editing Core has three central aims: 1) to assess on-target editing activity across all Research Projects from isolated cell lines and from animal tissue samples; 2) to assess off-target editing activity in treated cells; 3) to optimize gene editing technologies for therapeutic targets. The Core will play an essential role in generating rigorous analyses of gene editing outcomes for the IND submission of our trailblazer project, coordinating closely with the Preclinical Mouse Model Core analyze samples from large-scale efficacy studies. Additionally, the Core will facilitate the maturation of the Follower Projects by continuing to develop the latest base editing and/or prime editing technologies in model systems representative of each Follower Project's therapeutic target. Synergy between the Core and the Follower Projects will enable the co-development of new genome editing tools that maximize on-target editing efficiencies, minimize undesirable gene editing byproducts and off-target editing events, and maximize compatibility with in vivo delivery methods of potential therapeutic relevance.

项目总结基因编辑核心