Gene Delivery Core

基因传递核心

基本信息

批准号：
10458594
负责人：
Jose A Cancelas
金额：
$ 9.75万
依托单位：
CINCINNATI CHILDRENS HOSP MED CTR
依托单位国家：
美国
项目类别：
财政年份：
2021
资助国家：
美国
起止时间：
2021-08-01 至 2026-07-31
项目状态：
未结题

来源：
https://reporter.nih.gov/project-details/10458594
关键词：
Adenovirus Vector Back Basic Science Biological Biological Assay Biological Phenomena Biology CRISPR library CRISPR/Cas technology Cells Cellular biology Clinic Clinical Clinical Research Clinical Trials Clustered Regularly Interspaced Short Palindromic Repeats Collaborations Communities Complex Consultations Core Facility Cyclic GMP DNA DNA Viruses Development Developmental Therapeutics Program Disease Ensure Evaluation Feasibility Studies Functional disorder Funding Agency Future Gammaretrovirus Gene Delivery Gene Expression Gene Transfer Gene-Modified Genes Genetic study Goals Guide RNA Hematological Disease Hematology Human Human Genome Human Resources Individual Infrastructure Institution Investigation Investigational Drugs Knock-out Laboratories Laboratory Research Lentivirus Lentivirus Vector Libraries Materials Testing Medical center Methodology Molecular Monitor Mus Pediatric Hospitals Phase Preparation Procedures Process Production Quality Control RNA Reproducibility Research Research Personnel Research Project Grants Resources Robotics Serotyping Services Source Spumavirus System Technology Testing Time Training Translating Translational Research Translations Ultracentrifugation Viral Vector adeno-associated viral vector base bench to bedside biological research clinically relevant cost delivery vehicle design early phase clinical trial experience gene therapy genetic manipulation genetically modified cells high throughput screening industry partner innovation interest knock-down knockout gene mouse genome new technology novel pre-clinical preclinical study research and development scale up screening small hairpin RNA small molecule therapeutic development therapeutic gene tool translational barrier translational pipeline vector

项目摘要

Abstract Investigation of the molecular and cellular pathophysiology for hematologic diseases requires rapid and low-cost access to vector preparations, and access to large libraries of vectors and production of vectors destined to high throughput screening. Further, the successful development and testing of novel gene therapies requires access to a translational vector “pipeline” which necessitates a close interaction between basic research, research vector and clinical vector manufacturing laboratories. Cincinnati Children’s Hospital Medical Center, a clinical and research institution of excellence, has developed outstanding cores to provide hematology investigators with vector support for biological studies for the genetic manipulation of cells and their translation to the clinic. This core has a track record of providing high quality “research-grade” and “clinical-scale” vector services to investigators at various stages of basic and translational research, ensuring expeditious translation to the clinic. The components of the Gene Delivery Core (GDC) that contribute to this effort are the Viral Vector Core and Vector Library Core. Each of these sub-cores are equipped with its own state-of-the-art research laboratories to allow rapid access to vector libraries and viral vector products, their evaluation in routine basic research lab studies and/or high throughput screens, and their translation to the clinic as the studies move through the translational “pipeline”. All of this effort is backed by a strong Institutional commitment to translational research and widely popular demands from users in the broad hematology field. Our objectives are to provide: (1) research scale and medium scale pilot and feasibility viral vector products and consultation on vector type and design, and (2) vector library core services including production of lentiviral shRNA and CRISPR gRNA libraries for high throughput screening. Our overall goal is to advance research and development in hematology via facilitating gene delivery, gene knock down, gene knockout or gene editing, to accelerate the rate of translation.

抽象的血液疾病的分子和细胞病理生理学研究需要快速和低成本获得载体制剂、大型载体库和载体生产注定要进行高通量筛选，并成功开发和测试新基因。治疗需要访问翻译载体“管道”，这需要密切的相互作用辛辛那提基础研究、研究载体和临床载体制造实验室之间的关系。儿童医院医疗中心是一家卓越的临床和研究机构，出色的核心为血液学研究人员提供生物学研究的载体支持该核心拥有提供细胞基因操作及其转化到临床的良好记录。为基础研究各个阶段的研究人员提供高质量的“研究级”和“临床规模”载体服务和转化研究，确保基因的成分迅速转化为临床。为这项工作做出贡献的交付核心 (GDC) 是病毒载体核心和载体库核心。其中一些子核心配备了自己最先进的研究实验室，可以快速访问对于载体库和病毒载体产品，它们在常规基础研究实验室研究中的评估和/或高通量筛选，以及随着研究的进展将其转化为临床所有这些努力都得到了机构对转化研究的坚定承诺的支持。我们的目标是提供：(1)。研究规模和中规模试点和可行性病毒载体产品以及载体类型咨询和设计，以及（2）载体库核心服务，包括慢病毒shRNA和CRISPR的生产我们的总体目标是推进高通量筛选的 gRNA 文库。通过促进基因传递、基因敲低、基因敲除或基因编辑来加速血液学翻译率。