Gene Transfer

Sloan-Kettering Inst Can Research, New York NY

Investigators

Linked publications, trials & patents

Abstract

The Gene Transfer and Somatic Cell Engineering Core (GTF) supports the preclinical translation and clinical implementation of gene transfer studies at MSKCC. The projects supported are highly dependent on achieving efficient gene transfer in primary cells, including hematopoietic progenitor cells, T lymphocytes and dendritic cells. In the upcoming grant cycle, the GTF will focus on the implementation of Phase l/ll clinical trials and on the development and optimization of clinical cell engineering processes. The specific alms of the GTF are to carry out: 1) Expansion and transduction of patient cells for clinical trials utilizing expanded and/or genetically modified cells; 2) Generation and characterization of high-titer producer cell clones, master cell banks (MCB); 3) Production and titration of 5 to 15 liter batches of clinical viral stocks in semi-closed systems; 4) Biosafety testing in cultured packaging cell clones (MCB), viral stocks and clinical specimen; 5) Monitoring of retroviral vector copy number and integration sites by LAM-PCR in patient cells; and 6) Cell banking, storage of viral stocks, plasmid DNA and clinical specimens. In addition, the GTF provides essential advisory and training functions for generation of research grade reagents. The GTF is a repository for numerous reagents and protocols that are made available to investigators at MSKCC. The centralization of cell transdudion, vector production and plasmid DNA manufacturing in the GTF decreases the cost of clinical development. It also ensures high quality, consistency and timely implementation of molecular and cellular processes, and their availability to all investigators at the Center both at the preclinical and clinical stages. Over the last funding period the GTF enabled the successful implementation of 7 clinical trials currently open for enrollment under 4 investigational new drug applications. The services of the GTF supported the research of 11 investigators in the past year. During the past grant period the Core contributed to 107 publications of researchers from 3 programs.