CORE--DNA VECTOR

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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.