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Gene Therapy for Familial Adenomatous Polyposis

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source: Springer


The adenomatous polyposis coli (APC) gene is mutated in the germline of patients with hereditary disease familial adenomatous polyposis (FAP) and also been implicated in the pathogenesis of sporadic colorectal cancers. The APC gene in FAP provides a reasonable candidate for a preventive gene therapy strategy, since APC mutations appear to be fundamental to the early stages of all types of colorectal carcinogenesis, whether familial or sporadic. It is not known whether replacement of normal copies of APC gene can prevent hyperplasia, development of adenomata and ultimately prevent or delay malignant change.

DOI: 10.1007/978-1-4615-5357-1_60

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