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Genome Editing and Biological Effects Testing Section

基因组编辑及生物效应检测组

基本信息

批准号：
10773478
负责人：
William Raymond Lagor
金额：
$ 47.07万
依托单位：
BAYLOR COLLEGE OF MEDICINE
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：
2023-12-01 至 2028-11-30
项目状态：
未结题

来源：
https://reporter.nih.gov/project-details/10773478
关键词：
Acceleration Adverse effects Alleles Animal Testing Animals Biodistribution Biological Cells Chemistry Chromosomal translocation Communities Consult Data Development Disease Disease model Enzyme-Linked Immunosorbent Assay Event Excision Fee-for-Service Plans Flow Cytometry Germ Cells Goals Health Histologic Histopathology Immune response Immunophenotyping Knockout Mice Medicine Methods Modeling Monitor Mus Mutation Organ Pathogenicity Phase Phenotype Preclinical Testing Procedures Protein Secretion Reagent Reporter Reporting Research Research Personnel Resources Rice Safety Serum Specificity System Testing Time Tissue Harvesting Tissues Toxic effect United States National Institutes of Health Universities Western Blotting Work automated image analysis base editing cell type central database college experience experimental study gene product genome editing insertion/deletion mutation interest mouse model next generation sequencing novel strategies novel therapeutic intervention novel therapeutics precision medicine prime editing programs repaired restoration somatic cell gene editing tool vector genome

项目摘要

3. ABSTRACT – GENOME EDITING AND BIOLOGICAL EFFECTS TESTING SECTION The goal of the Genome Editing and Biological Effects Testing Section (GE-BETS) of the BCM/Rice Genome Editing Testing Section is to provide to the research community a centralized, broadly accessible, fee-for-service resource for testing genome editing delivery reagents and genome editing tools in mouse models. The GE-BETS will develop new approaches and standard operating procedures (SOPs), as well as customize analyses based on research needs. GE-BETS capitalizes on extensive local genome editing and testing experience within Baylor College of Medicine (BCM) and Rice University, originating from the BCM Knockout Mouse Phenotyping Project, Center for Precision Medicine Models, and the BCM-Rice Small Animal Testing Center of the NIH Somatic Cell Genome Editing program. In this project, GE-BETS will develop pipelines for testing of genome editing reagents in genome editing reporter and disease models and perform testing for external research partners. In Aim 1, we will perform testing of somatic genome editing systems in fluorescent reporter mice. Major deliverables include biodistribution, editing efficiency across tissues, cell type identification, and changes in health or toxicity arising from genome editing and delivery systems. In Aim 2, we will determine the efficiency, specificity, and safety of genome editing reagents in relevant disease models. We will assess on- and off-target editing by next generation sequencing in the target tissue to precisely quantify the efficiency of genome editing in the context of a pathogenic mutation. Editing efficiency will be correlated with restoration or removal of the intended gene product. These studies will inform the feasibility of new therapeutic approaches, as well as identify unintended adverse effects of genome editing. Consolidating state of the art mouse models and expertise under a fee-for- service core, will broaden access to high quality testing data and accelerate the development of new therapeutics.

3. 摘要-基因组编辑与生物效应测试部分