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.
| Saetrom, Pål; Biesinger, Jacob; Li, Sierra M et al. (2009) A risk variant in an miR-125b binding site in BMPR1B is associated with breast cancer pathogenesis. Cancer Res 69:7459-65 |
| Kowolik, Claudia M; Yam, Priscilla; Yu, Ying et al. (2003) HIV vector production mediated by Rev protein transduction. Mol Ther 8:324-31 |
| Li, Ming-Jie; Bauer, Gerhard; Michienzi, Alessandro et al. (2003) Inhibition of HIV-1 infection by lentiviral vectors expressing Pol III-promoted anti-HIV RNAs. Mol Ther 8:196-206 |
| Paul, Cynthia P; Good, Paul D; Li, Shirley X L et al. (2003) Localized expression of small RNA inhibitors in human cells. Mol Ther 7:237-47 |
| Strayer, David; Branco, Francisco; Zern, Mark A et al. (2002) Durability of transgene expression and vector integration: recombinant SV40-derived gene therapy vectors. Mol Ther 6:227-37 |
| Lee, Nan Sook; Dohjima, Taikoh; Bauer, Gerhard et al. (2002) Expression of small interfering RNAs targeted against HIV-1 rev transcripts in human cells. Nat Biotechnol 20:500-5 |
| Yam, Priscilla Y; Li, Shulian; Wu, Jerry et al. (2002) Design of HIV vectors for efficient gene delivery into human hematopoietic cells. Mol Ther 5:479-84 |
| Michienzi, Alessandro; Li, Shirley; Zaia, John A et al. (2002) A nucleolar TAR decoy inhibitor of HIV-1 replication. Proc Natl Acad Sci U S A 99:14047-52 |
| Chang, Zongli; Westaway, Shawn; Li, Shirley et al. (2002) Enhanced expression and HIV-1 inhibition of chimeric tRNA(Lys3)-ribozymes under dual U6 snRNA and tRNA promoters. Mol Ther 6:481-9 |
| Michienzi, A; Rossi, J J (2001) Intracellular applications of ribozymes. Methods Enzymol 341:581-96 |
Showing the most recent 10 out of 12 publications
