A paradigm for cancer treatment using the retinoblastoma gene in a mouse model.
Discovery of tumor suppressor genes has provided a rational approach to cancer prevention and treatment. Loss of retinoblastoma susceptibility gene (Rb) function is a rate-limiting event in the development of human and mouse cancers. Establishment of animal models of cancer associated with Rb deficiency allowed us to develop and test long-awaited approaches to genetic correction for treating tumors in vivo. Recent studies demonstrated that (1) prevention of carcinogenesis is achieved by correction of gene copy number in Rb+/- mice, and (2) reconstitution of Rb gene functions is sufficient for suppression of neoplasia in immunocompetent mice. These results fulfill a promise of cancer treatment by reconstitution of tumor suppressor function.
Nikitin
A Y
AY
Department of Molecular Medicine/Institute of Biotechnology, University of Texas Health Science Center, San Antonio 78245-3207, USA.
Riley
D J
DJ
Lee
W H
WH
D000818:Animals; D004195:Disease Models, Animal; D016161:Genes, Retinoblastoma; D015316:Genetic Therapy; D006801:Humans; D051379:Mice; D009374:Neoplasms, Experimental / Q000235:genetics* / Q000628:therapy