Gene Transfer and Somatic Cell Enginerring

ABSTRACT The mission of the Gene Transfer and Somatic Cell Engineering (GTSC) Core is to develop, validate, and supply cell-based experimental products for Phase I and II clinical trials so that investigators can evaluate their safety and potential clinical benefit. The Core supports the implementation of Phase I/II clinical trials based on cell engineering and adoptive cell therapies. Its major goals are as follows: 1) To develop SOPs and batch records for cell processing and engineering, vector production; 2) GMP manufacturing: Patient cell (expansion, purification, transduction, formulation); Vector stocks (plasmid DNA, cell banks); 3) biosafety/release testing: Patient cells and vector stocks (e.g., phenotype, potency, purity, stability); Gene transfer/expression; 4) to monitor patients’ samples post-infusion (e.g., FACS, Q-PCR); and 5) to provide technological and regulatory expertise (GXP, FDA, RAC-NIH).