CORE--DNA VECTOR

DNA载体核心产生并表征所有病毒和非病毒的 临床前和临床项目所需的病毒DNA载体。基于 在12，000平方米的英尺Belfer基因治疗核心设施，核心包括 新装修、设备齐全的研究区和2400平方米的英尺 临床（药品生产质量管理规范[GMP]）生产设施。 GMP设施的设计和建造符合所有 FDA对制备用于以下疾病的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 设施和设备的监测和人员，材料， 废物和空气流。运行一个完全符合GMP的设施是超越 任何小组的手段和共享这种最先进的设施 在NHLBI之后，与Weill Cornell PEGT和其他PEGT研究人员一起 审查是对这一宝贵资源的有效利用。临床 (GMP)设施将由斯蒂芬·卡明斯基博士领导。并将 为项目6提供媒介，并为试验性研究提供设施， 涉及离体的项目2和4的临床前开发方面 将基因转移到打算再次施用的患者细胞中。临床 (GMP)工作人员还将与PEGT调查人员密切合作， DNA载体核心的临床前臂，以改善 DNA载体的纯化和表征。临床前DNA 矢量核心，导演尼尔哈克特博士，将提供腺病毒， AAV和质粒及脂质复合物作为项目2、3和4的载体 并根据需要进行合作，采用新的基因技术， 随着他们的出现而转移。收集方案和生物材料 还可以使用描述性数据库来加快 PEGT项目。核心小组将参与联合国教育、科学及文化组织的教育工作， PEGT和培养博士后研究员在载体技术 生产和表征。

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.