Although the first successful human gene therapy was performed in XSCID patients, the occurrence of T cell leukemia in four of ten successfully treated patients three to six years following treatment has shown that gene therapy is not without potential serious adverse side effects. In this application we will use the unique canine XSCID model as a pre-clinical large and long-lived animal model to test new vectors and strategies for improving the efficacy and durability of retroviral gene therapy prior to treating human patients.
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