by Carl F. Perez, PhD, Sandra L. Vanderbyl, Kathleen A. Mills, PhD, Harry C. Ledebur, Jr., PhD
Volume 3, Issue 4 (July/August 2004)
Current in vivo gene therapy (GT) approaches are beginning to demonstrate significant clinical and safety limitations that may ultimately reduce their therapeutic utility. In particular, the potential for systemic toxicity due to the antigenicity of the gene transfer vector, the prospect of insertional mutagenesis/oncogenesis during gene transfer, and the possibility of germ line transfer of the transgene are issues raising concern. One promising alternative to gene therapy that mitigates these clinical and safety issues is gene-based cell therapy (GBCT), in which autologous cells are removed from a patient and modified ex vivo for a desired characteristic prior to reimplantation. By transferring the transgene ex vivo, many of the issues surrounding the in vivo use of the transfer vectors are reduced and issues surrounding germ line transfer can be practically eliminated…
Citation:
Perez CF, Vanderbyl SL, Mills KA, Ledebur HC. The ACE System: A Versatile Chromosome Engineering Technology with Applications for Gene-Based Cell Therapy. BioProcess J, 2004; 3(4): 61-68.