Gene Therapy

The Belfer Gene Therapy Core Facility provides the infrastructure to carry out basic, translational and clinical research utilizing gene transfer.

Facilities

The Belfer Gene Therapy Core Facility is a new, fully equipped core facility devoted exclusively to developing and assessing gene transfer vectors. The Vector Core includes 6 independent cell culture rooms plus bench and desk space for 8 scientists, an office for the Director and an instrument room containing large equipment. The Director supervises a technical staff of 9 (7 technicians and 2 postdoctoral associates) individuals. The Vector Core includes analytical resources: quantitative PCR, 2 HPLC's, plate readers, automated liquid handling devices, phosphor-/fluoro-imager and appropriate computer support and backup. There is also a database of the available vectors and plasmids with extensive sequence and restriction mapping data. The Good Manufacturing Practice (GMP) Core is comprised of the GMP facility which occupies approximately 2,400 square feet devoted exclusively to production of gene transfer vectors and gene modified cells for human therapeutic trials. There is office space and supporting laboratory space for analyses and quality control of GMP grade vectors including a room with a Biostat C fermentor. The design, construction, and validation of the GMP production facility is monitored and reviewed with the FDA to assure full compliance with all current GMPs. The GMP facility consists of a suite of three fully equipped production rooms, a staging area, gowning and degowning pathways laid out with a series of interlocking double-door pass-through devices and doorways. In conjunction with carefully maintained differential room pressure relationships and single pass HEPA filter air, standard operating procedures define the flow of material, personnel, and waste through the facility and particulates and equipment are monitored full time. In addition, there are separate rooms for QC released and quarantined materials, a cell bank and a quality control lab. There is an extensive set of standard operating procedures for the use, cleaning, and validation of equipment, and for the purchase, tracking, and storage of supplies and for training personnel.

Services

The Vector Core functions as a resource to investigators to provide centralized expert services and training in the design, creation, and production of gene transfer vectors, and to provide a characterized repository of gene transfer vectors and related reagents for use by investigators. The primary resource of the Vector Core is the expertise to efficiently design, construct and produce gene transfer vectors, including adenovirus (Ad), adenoassociated virus (AAV), lentivirus, retrovirus vectors, as well as non-viral vectors and plasmid vectors. New cDNA's of biological significance are continuously being acquired and used to make first generation and more advanced gene transfer vectors. The Vector Core maintains, and continually updates, standard operating procedures for the construction, production, and verification of gene transfer vectors. Many individual steps of vector production have been optimized and new protocols and reagents are evaluated and incorporated into the standard operating procedures.