Gene Delivery Core

基因传递核心

基本信息

批准号：
10201889
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/10201889
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 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 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.

抽象的研究血液学疾病的分子和细胞病理生理学需要快速，并且需要快速低成本访问矢量准备，并访问大型向量和媒介的生产注定要进行高吞吐量筛查。此外，新基因的成功开发和测试疗法需要访问翻译向量“管道”，这涉及紧密的互动在基础研究，研究载体和临床向量制造实验室之间。辛辛那提儿童医院医疗中心是一家卓越临床和研究机构，已发展杰出的核心为血液学研究者提供为生物学研究的载体支持细胞的基因操纵及其转化为诊所。该核心有提供的记录高质量的“研究级”和“临床规模”向量服务为研究人员的基本各个阶段并翻译研究，确保迅速转化为诊所。基因的成分促成这项工作的交付核心（GDC）是病毒矢量核心和矢量库核心。每个这些子核心配备了自己的最先进的研究实验室，以允许快速访问对于向量库和病毒矢量产品，它们在常规基础研究实验室研究和/或高吞吐量屏幕及其转化为诊所，随着研究通过翻译 “管道”。所有这些努力都得到了对翻译研究和广泛血液学领域的用户的广泛受欢迎。我们的目标是提供：（1）研究量表和中等规模的飞行员和可行性病毒向量产品以及矢量类型的咨询和设计，以及（2）矢量库核心服务，包括生产慢病毒shrna和crispr GRNA库，用于高通量筛选。我们的总体目标是进步通过促进基因递送，基因敲除，基因敲除或基因编辑的血液学，以加速翻译速率。