| First Author | Goebel WS | Year | 2004 |
| Journal | Blood Cells Mol Dis | Volume | 33 |
| Issue | 3 | Pages | 365-71 |
| PubMed ID | 15528159 | Mgi Jnum | J:95009 |
| Mgi Id | MGI:3522514 | Doi | 10.1016/j.bcmd.2004.06.007 |
| Citation | Goebel WS, et al. (2004) Stable long-term gene correction with low-dose radiation conditioning in murine X-linked chronic granulomatous disease. Blood Cells Mol Dis 33(3):365-71 |
| abstractText | We previously demonstrated that low-dose radiation conditioning impairs murine hematopoietic stem cell function, permitting engraftment of syngeneic fresh and transduced marrow cells. In this study, we directly examined the ability of low-dose radiation conditioning to permit engraftment of transduced long-term repopulating cells in murine X-linked chronic granulomatous disease (X-CGD), which closely mimics the human disease. X-CGD mice conditioned with 160 cGy were transplanted with 20 x 10(6) MSCV-m91Neo-transduced syngeneic X-CGD marrow cells. The presence of oxidase-positive neutrophils in two independent cohorts of transplanted 160-cGy-conditioned X-CGD recipients was determined by nitroblue tetrazolium testing. Transplanted X-CGD mice (n = 9 total) displayed 1-17% oxidase-positive neutrophils 6-16 months post-transplant. Retroviral marking and NADPH-oxidase-positive neutrophils persisted through serial transplantation, verifying that stem cells were transduced. These results establish that low-dose radiation conditioning results in durable engraftment of low but potentially clinically relevant numbers of functionally reconstituted blood cells in a murine model of X-CGD. |
