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BRAIN Viral Vector Services and Distribution Core

BRAIN 病毒载体服务和分发核心

基本信息

批准号：
10685426
负责人：
Kimberly Ritola
金额：
$ 39.79万
依托单位：
UNIV OF NORTH CAROLINA CHAPEL HILL
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：
2021-09-01 至 2026-08-31
项目状态：
未结题

来源：
https://reporter.nih.gov/project-details/10685426
关键词：
Animals BRAIN initiative Biological Sciences Biology Biomanufacturing Biotechnology Brain Brain Diseases Capsid Cell Therapy Client Collaborations Communities Country Custom Dependovirus Development Educational workshop Equipment Genome Goals Human Resources International Letters Maintenance Medical Mission Neurodegenerative Disorders Neurodevelopmental Disorder Neurological Models Neurosciences North Carolina Performance Preparation Privatization Production Protocols documentation Quality Control Rabies Rabies virus Ramp Reagent Research Research Personnel Resources Services Source Synapses System Training and Education Translating Universities Validation Vendor Viral Viral Vector Virus Work adeno-associated viral vector base brain cell brain research cell type cost design education resources experience experimental study gene replacement gene therapy improved in vivo manufacture nervous system disorder neural circuit neuronal circuitry pre-clinical promoter rabies viral tracing recombinase repository sensor skills success tool translational study vector

项目摘要

The BRAIN Viral Vector Service and Distribution Core will serve as a resource for the maintenance, propagation, and distribution of viral vectors that are used by neuroscientists for neural circuit identification and manipulation, as well as for preclinical translational studies. High-quality viral vectors are essential tools to mark and manipulate neuronal circuits in live animals and identify long- range synaptic connections. Moreover, adeno-associated virus (AAV) vectors are the de facto standard for gene therapies. Existing commercial viral vector sources do not offer end-to-end services, from assisting with viral vector design, rigorous manufacturing and quality controls, troubleshooting with clients to enhance vector performance in the brain, and a mission focused on neuroscience. The Core Director (Dr. Kim Ritola) will translate her ten years of providing viral tools to the Howard Hughes Medical Institute neuroscience community towards creating a BRAIN Viral Vector Service and Distribution Core capable of producing and distributing AAV, rabies, and lenti virus tools to neuroscientists. The Core will have two main purposes: 1) Produce and disseminate affordable, high-quality AAV, rabies, and lenti viral vectors to the neuroscience community including newly developed vectors, custom preps and curated stocks and 2) Collaborate with BRAIN researchers to optimize vector design and prep conditions for optimal performance and serve as an educational resource for viral vector production. This Core will provide investigators with an extensive choice of vector capsid or envelope, paired with custom viral construct, including those with traditionally difficult to package genomes, titers and volumes appropriate for experiment type, rigorous quality controls, and affordability.

大脑病毒载体服务和分发核心将作为维护的资源，神经科学家用于神经回路的病毒载体的传播和分布鉴定和操作，以及临床前转化研究。高质量的病毒载体是标记和操纵活体动物神经回路的基本工具，突触连接的范围此外，腺相关病毒（AAV）载体是事实上的病毒载体。基因治疗的标准。现有的商业病毒载体来源不提供端到端的服务，从协助病毒载体设计，严格的生产和质量控制，与客户进行故障排除，以增强大脑中的向量性能，以及一项使命，重点是神经科学核心总监（Kim Ritola博士）将把她十年来提供病毒工具的经验转化为霍华德休斯医学研究所神经科学社区致力于创造一种脑病毒载体能够生产和分销AAV、狂犬病病毒和慢病毒工具的服务和分销中心神经科学家。核心将有两个主要目的：1）生产和传播负担得起的，高质量的AAV、狂犬病病毒和慢病毒载体，开发载体，定制准备和策划股票和2）与大脑研究人员合作，优化载体设计和准备条件，以实现最佳性能，并作为教育用于病毒载体生产的资源。该核心将为研究者提供广泛的选择，载体衣壳或包膜，与定制的病毒构建体配对，包括那些传统上难以包装适合实验类型的基因组、滴度和体积，严格的质量控制，和可负担性。