CORE--DNA VECTOR

DNA载体核心产生并表征了所有的病毒和非 临床前和临床项目所需的病毒DNA载体。基座 在12,000平方英尺的英国《金融时报》贝尔弗基因治疗核心设施，核心包括 一个新装修的设备齐全的研究区和2400平方米。英国《金融时报》 临床(良好生产规范[GMP])生产设施。 GMP工厂的设计和建造符合所有 FDA对制造载体和体外细胞疗法的要求 人类。功能包括设施和设备验证、连续 设施和设备监测和控制人员、材料、 废物和空气流动。运行完全合规的GMP设施超出了 任何小团体的手段和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 审查代表了对这一宝贵资源的有效利用。临床部 (GMP)设施将由Stephen Kaminsky博士指导，并将 为项目6提供载体，并为试点研究提供设施 项目2和项目4涉及体外的临床前发展方面 将基因转移到患者细胞以供管理。临床部 (GMP)人员还将与PEGT调查人员和 临床前臂DNA载体核心全面提升 DNA载体的纯化和鉴定。临床前DNA 由尼尔·哈克特博士指导的载体核心公司将提供腺病毒， AAV和质粒及脂类复合体作为项目2、3和4的载体 以及根据需要进行协作，以采用新的基因技术 在它们出现时转移。协议和生物材料的集合 还提供了描述性数据库，以加快 PEGT项目。中心将参与教育方面的工作 PEGT和培训矢量技术博士后 制作和刻画角色。

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.