This Core will serve as the central PCR diagnostic laboratory to evaluate the efficacy of the different strategies implemented in the scientific projects (Projects 1-3) and to monitor the overall progress of the clinical trial (Project). This will be accomplished by verifying the transduction efficiency of stem cells; establishing the presence of an active expression of the therapeutic gene in these cells; detecting the presence and long- term expression of the transduced retroviral vectors in patient samples, and by evaluating the antiviral activity of the ribozyme in conjunction with tests done in Project 2. Importance of Core to IPCP Program. The critical endpoint determination, namely the presence of ribozyme transgene in transduced cells, will be determined by this Core for the scientific projects and for the two gene therapy trials.
| 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 |
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