Gene Therapy for Familial Adenomatous Polyposis
Corrective gene therapy for the treatment of single gene disorders has reached the stage of clinical trials. For malignant disease however research and clinical gene therapy protocols have focused on using genetic techniques to enhance the host’s immune response to a tumour or to direct cytotoxic therapy to tumour cells [1–3]. Corrective gene therapy has been largely ignored as a potential strategy for the treatment of malignant disease due to perceived difficulties in achieving an adequate cell hit rate [4, 5], However recent clinical  and preclinical studies [7, 8] have begun to address the problems raised by this strategy. Genetic modifications which reverse the malignant process could however lead to negative selection pressure followed by overgrowth of residual unmodified cancer cells. It would therefore be preferable if tumour suppressor genes could be introduced into normal tissue at a high risk of becoming malignant in order to prevent transformation, rather than reverse the neoplastic process.
KeywordsGene Therapy Familial Adenomatous Polyposis Adenomatous Polyposis Coli Adenomatous Polyposis Coli Gene Intestinal Stem Cell
adenomatous polyposis coli
familial adenomatous polyposis
murine intestinal neoplasia
polymerase chain reaction
- RT PCR
reverse transcriptase polymerase chain reaction
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