Gene Transfer into the Colonic Mucosa

Somatic gene therapy is based on the principle of transferring recombinant genes efficiently into somatic tissues and achieving expression of the gene product in order to replace genetically defective gene functions or alter pathological disease processes. The development of a gene therapy model system that can stably produce and deliver bioactive target proteins into the intestinal microenvironment may represent an important advance in the treatment of several gut-related diseases including inflammatory bowel disease (IBD) and colon cancer. Ideally, transfection of the gut epithelia and their progenitor stem cells (i.e., epithelial crypt cells), would enable the local and targeted production of the desired gene product into the intestinal milieu. Furthermore, such genetically altered cells would have the ability to replicate the transfected gene and continue to produce and secrete its specifically encoded protein without interfering with the function of the tissue in which they reside.