Shared Resources Core-Viral Vector Core

共享资源核心-病毒载体核心

• 批准号: 10712151
• 负责人: JOEL R CHAMBERLAIN
• 金额: $ 15.89万
• 依托单位: UNIVERSITY OF WASHINGTON
• 依托单位国家: 美国
• 财政年份: 2014
• 资助国家: 美国
• 起止时间: 2014-05-07 至 2028-08-31
• 项目状态: 未结题
• 来源: https://reporter.nih.gov/project-details/10712151
• 关键词: Animal Model; Animals; Antibodies; Basic Science; Biotechnology; Canis familiaris; Capsid; Cardiac Myocytes; Cell Line; Cells; Centrifugation; Charge; Clinical Research; Collaborations; Complementary DNA; Consumption; Cytokeratin-8 Staining Method; Cytomegalovirus; Doctor of Philosophy; Dystrophin; Family suidae; Funding; Gene Delivery; Gene Transfer; Genes; Hand; High Pressure Liquid Chromatography; Human Resources; Immune Sera; Laboratories; Learning; Lentivirus Vector; Medical; Methods; Muscle; Muscular Dystrophies; Myoblasts; Myopathy; Plasmid Cloning Vector; Plasmids; Play; Preparation; Procedures; Production; Protein Isoforms; Protocols documentation; Quality Control; RNAi vector; Reagent; Recombinants; Regulator Genes; Research; Research Personnel; Resource Sharing; Resources; Retroviral Vector; Role; Roller Bottle; Serotyping; Services; Techniques; Technology; Time; Training; Translational Research; Universities; Utrophin; Vertebral column; Viral; Viral Vector; Visit; Washington; Work; adeno-associated viral vector; cost; design; gene therapy; improved; induced pluripotent stem cell; interest; member; mouse model; prevent; research study; stem cells; vector; web site

Project Summary – Resource (Vector) Core The Viral Vector Core was established in 2003 to meet the emerging interest in using AAV & lentiviral vectors for muscle gene transfer. The required specialized techniques and reagents prevented most labs from being able to generate the high titer and enormous quantities of vectors needed for systemic gene delivery, challenges that remain today. Consequently, a Viral Vector Core was established to generate vectors for Wellstone Centers, but which has been expanded to allow access to a broader range of users via a recharge mechanism. This Vector Core is our Scientific Research Resource Core. The Core provides high quality preparations of viral and plasmid vectors to facilitate research related to the muscular dystrophies. Services offered by the Core include vectors, reagents, consulting, and training related to vector production. This includes production of adeno- associated and lentiviral vectors and muscular dystrophy related reagents (cell lines, vector backbones, protocols, antibodies etc) for members of this Center as well as laboratories in the US and across the World to support their efforts related to gene transfer and gene therapy for the muscular dystrophies. The Principle Investigator is Dr. Joel Chamberlain, who has worked closely with Core personnel on AAV and retroviral vectors for more than 15 years. The Core has established protocols and acquired reagents for lentiviral and adeno- associated viral vectors, particularly AAV6, 8 and 9, but also for other pseudotyped vectors including AAV1, 2, 5, 7 and several recently identified myotropic (e.g. AAVMyo) capsids. We also have numerous muscle cDNAs, such as for dystrophin and utrophin, muscle regulatory cassettes and antibodies that are available via this Core lab, as well as DM1, DUX4 and associated RNAi vectors. We provide high quality, high titer preparations of AAV and lentiviral vectors and also encourage users to visit the Core to learn, or request, our methods. Vectors and reagents are provided to members of our Wellstone Center using funds from this Core, and to other muscular dystrophy researchers on a recharge basis at cost. Training services allow investigators to apply methods for production and purification of viral vectors in their own labs as desired, and will be advertised via the Center Web site. Consulting will be available to guide the use of AAV and lentiviral vectors. Finally, we provide reagents (such as vector backbones, plasmids, antibodies) that have been developed by members of this Wellstone Center to outside labs.

项目摘要 - 资源（向量）核心 病毒矢量核心于2003年成立，以满足使用AAV和慢病毒载体的新兴兴趣 用于肌肉基因转移。所需的专业技术和试剂阻止了大多数实验室 可以产生全身基因传递所需的高滴度和大量向量，挑战 今天仍然如此。因此，建立了病毒矢量核心，以生成井石中心的向量， 但是，已经扩展了，以允许通过充电机制访问更广泛的用户。这 向量核心是我们的科学研究资源核心。核心提供了病毒和高质量的准备工作 质粒向量促进与肌肉营养不良有关的研究。核心提供的服务包括 与矢量生产有关的媒介，试剂，咨询和培训。这包括生产adeno- 相关和慢病毒载体以及肌肉营养不良相关试剂（细胞系，载体骨架， 该中心成员以及美国以及世界各地的实验室的协议，抗体等） 支持他们与肌肉营养不良的基因转移和基因治疗有关的努力。原则 调查员是乔尔·张伯伦（Joel Chamberlain）博士，他曾与AAV和逆转录病毒载体紧密合作 超过15年。核心已经建立了方案并为慢病毒和腺体获得了试剂 相关的病毒矢量，特别是AAV6、8和9，但也适用于其他假型向量，包括AAV1、2， 5、7和几个最近确定的肌体（例如Aavmyo）衣壳。我们也有许多肌肉cDNA， 例如肌营养不良蛋白和乌托蛋白，可通过此核心提供的肌肉调节盒和抗体 实验室以及DM1，DUX4和相关的RNAi矢量。我们提供AAV的高质量，高滴度的准备 和慢病毒向量，还鼓励用户访问核心以学习或要求我们的方法。向量和 使用该核心和其他肌肉的资金向我们的Wellstone中心成员提供试剂 营养不良的研究人员以成本为基础。培训服务使调查人员可以应用方法 根据需要在自己的实验室中生产和纯化病毒向量，并将通过中心网络进行广告 地点。咨询将用于指导AAV和慢病毒载体的使用。最后，我们提供试剂（这样 作为载体骨架，质粒，抗体），该中心的成员已经开发 外部实验室。