CORE--DNA VECTOR

核心--DNA载体

• 批准号: 6668359
• 负责人: Neil R. Hackett
• 金额: $ 19.94万
• 依托单位: WEILL MEDICAL COLL OF CORNELL UNIV
• 依托单位国家: 美国
• 财政年份: 2002
• 资助国家: 美国
• 起止时间: 2002-09-01 至 2003-08-31
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/6668359
• 关键词: biomedical facility; biotechnology; cooperative study; gene therapy; transfection /expression vector

The DNA Vector Core produces and characterizes all the viral and non- viral DNA vectors required by the preclinical and clinical projects. Based in the 12,000 sq. ft. Belfer Gene Therapy Core Facility, the Core consists of a newly renovated, fully equipped research area and a 2400 sq. ft. Clinical (Good Manufacturing Practice [GMP]) manufacturing facility. The GMP facility has been designed and constructed to comply with all FDA requirements for making vector and ex vivo cell therapies for humans. Features include facility, and equipment validation, continuous facility and equipment monitoring and control of personnel, material, waste and air flow. Running a fully compliant GMP facility is beyond the means of any small group and the sharing of this state of the art facility with Weill Cornell PEGT and other PEGT investigators after NHLBI review represents an efficient use of this valuable resource. The clinical (GMP) facility will be directed by Stephen Kaminsky Ph.D. and will provide vector for project 6 and a facility for pilot studies in the preclinical development aspects of projects 2 and 4 involving ex vivo gene transfer to patient cells intended for readministration. The clinical (GMP) personnel will also work closely with the PEGT investigators and preclinical arm of the DNA vector core to improve all aspects of purification and characterization of DNA vectors. The preclinical DNA Vector Core, directed by Neil Hackett Ph.D.., will provide adenoviral, AAV and plasmid and lipid complexes as vectors for projects 2, 3 and 4 as well as collaborate as required to adopt new technologies for gene transfer as they emerge. Collections of protocols and biological materials with a descriptive database are also available to expedite progress in the PEGT projects. The Core will participate in the education aspects of the PEGT and train post-doctoral fellows in the technology of vector production and characterization.

DNA 载体核心产生并表征所有病毒和非病毒 临床前和临床项目所需的病毒DNA载体。基于 在 12,000 平方英尺的 Belfer 基因治疗核心设施中，核心设施包括 一个新装修的、设备齐全的研究区和一个 2400 平方英尺的研究区。 临床（良好生产规范 [GMP]）生产设施。 GMP 设施的设计和建造符合所有 FDA 对载体和离体细胞疗法的要求 人类。功能包括设施和设备验证、连续 人员、物资、设施设备监测与控制 废物和空气流动。运行完全符合 GMP 设施的要求超出了 任何小团体的手段以及共享这种最先进的设施 NHLBI 后与威尔·康奈尔 PEGT 和其他 PEGT 调查人员合影 审查代表了对这一宝贵资源的有效利用。临床上 (GMP) 设施将由 Stephen Kaminsky 博士领导。并将 为项目 6 提供载体并为试点研究提供设施 项目 2 和 4 涉及离体的临床前开发方面 将基因转移到用于重新给药的患者细胞。临床上 (GMP) 人员还将与 PEGT 研究人员密切合作， 临床前臂DNA载体核心各方面改进 DNA载体的纯化和表征。临床前DNA Vector Core 由 Neil Hackett 博士指导，将提供腺病毒、 AAV 以及质粒和脂质复合物作为项目 2、3 和 4 的载体 以及根据需要进行合作，采用基因新技术 当它们出现时转移。协议和生物材料的集合 还可以使用描述性数据库来加快进展 PEGT 项目。核心将参与教育方面的工作 PEGT并培养载体技术博士后 生产和表征。