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Discovery In "Bubble Boy" Disease Gene Therapy

due to gene therapy of other blood disorders is an important piece of good news that comes out of this study. "If we could find a way to eliminate the excess cells, and the ability of gamma C to insert into oncogenes and turn them on, we could substantially reduce the risk of gene therapy for XSCID as well," Sorrentino said. He and his colleagues at St. Jude are currently studying ways to "insulate" genes used for gene therapy in order to prevent them from inserting themselves into oncogenes and turning them on. The St. Jude XSCID mouse models will then be used to test these new vectors.

The (Arf -/- gamma C -/- ) mouse models were developed using Arf -/- mice provided by Charles Sherr, M.D., Ph.D., co-chair of the St. Jude Department of Genetics and Tumor Cell Biology and a Howard Hughes Medical Institute investigator. Other authors of the paper include Zhijun Ma and Taihe Lu.

Source: Eureka Alert


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