The goal of the Clinical Trials Center is the development of a method for genetic therapy of HIV-1 using gene modified stem cells transplantation (SCT). This consists of a consortium between City of Hope National Medical Center and the University of Southern California School of Medicine (USC), including both Children's Hospital LA (Project 1) and Los Angeles County-USC Medical Center.
The specific aims of the project are: 1) To complete the follow-up of AIDS subjects transplanted for non- Hodgkin's lymphoma (NHL) using gene modified peripheral blood progenitor cell (PBPC); 2) To initiate an experimental autologous SCT model in HIV-1 infected persons using partial marrow cytoablation with hydroxyurea followed by transplantation with PBMC. In both these studies, PBMC will have been transduced with a retrovirus which encodes ribozymes targeted to TAT and REV. This will permit the comparison of a partial-ablation conditioning regimen for duration of expression of the transgene in the blood and marrow of the recipient. The information learned from these clinical trials and from the basic science projects within this program will be important for design of future gene therapy trials for AIDS. Thus, this project is important to the overall program because it is the center for all clinical evaluations of the anti- HIV gene therapy strategies under investigation in the program.
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