| First Author | Themis M | Year | 2005 |
| Journal | Mol Ther | Volume | 12 |
| Issue | 4 | Pages | 763-71 |
| PubMed ID | 16084128 | Mgi Jnum | J:102125 |
| Mgi Id | MGI:3606835 | Doi | 10.1016/j.ymthe.2005.07.358 |
| Citation | Themis M, et al. (2005) Oncogenesis following delivery of a nonprimate lentiviral gene therapy vector to fetal and neonatal mice. Mol Ther 12(4):763-71 |
| abstractText | Gene therapy by use of integrating vectors carrying therapeutic transgene sequences offers the potential for a permanent cure of genetic diseases by stable vector insertion into the patients' chromosomes. However, three cases of T cell lymphoproliferative disease have been identified almost 3 years after retrovirus gene therapy for X-linked severe combined immune deficiency. In two of these cases vector insertion into the LMO2 locus was implicated in leukemogenesis, demonstrating that a more profound understanding is required of the genetic and molecular effects imposed on the host by vector integration or transgene expression. In vivo models to test for retro- and lentiviral vector safety prior to clinical application are therefore needed. Here we present a high incidence of lentiviral vector-associated tumorigenesis following in utero and neonatal gene transfer in mice. This system may provide a highly sensitive model to investigate integrating vector safety prior to clinical application. |
