June 10, 2004
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Mouse model of retinoblastoma may aid in drug development

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Proposed therapies for retinoblastoma may be tested in the laboratory now for the first time, with the development of a mouse model that closely mimics the eye cancer, according to researchers at St. Jude Children’s Research Hospital.

Michael Dyer, PhD, and colleagues at St. Jude’s developed a “knockout” mouse model, so called because the gene Rb1 and two other cancer-suppressing genes have been eliminated in these animals. Rb1 deficiency has been identified as a genetic precursor to developing retinoblastoma. Previous attempts to develop a mouse model were unable to eliminate all three genes.

Dr. Dyer said that, because the tumor-suppressor genes are knocked out only in the retinas of these developing mice embryos, the mice do not develop other cancers and can therefore be bred to pass on the mutation.

“Only about 300 children develop retinoblastoma in the United States each year,” Dr. Dyer said. “It’s very difficult to gather enough children in one place to conduct a clinical trial of potential new anti-cancer drugs.”

A study describing the model is published in the June issue of Cell Cycle.