Table 1.
Summary of vector integration site sequence analysis.
| Animal ID | Infused CD34+ HSPCs (Marking)a | Cell Typesb | Months | Uniq. VIc | QVI (LT-QVI)d | Genom. Sitese |
|---|---|---|---|---|---|---|
| 95E132§ | 36×106 (20%; 7×106 GFP+) | Serial PBC (12) | 4 – 145 | 803 | 492 (233) | 726 |
| CD4, CD8, CD20, CD14, CD18, CD34 | 116 – 117 | 399 | 259 (259) | 365 | ||
| Total | - | 846 | 517 (259) | 759 | ||
| 2RC003§* | 30×106 (30%; 9×106 GFP+) | Serial Grans (9) | 3 – 103 | 679 | 475 (229) | 562 |
| Serial PBMC (9) | 3 – 103 | 607 | 425 (210) | 508 | ||
| CD4, CD8, CD20, CD14, CD18, CD34 | 70 – 71 | 360 | 279 (279) | 282 | ||
| Total | - | 938 | 614 (279) | 737 | ||
| RQ5427¶ | 160×106 (7%; 11×106 GFP+) | Serial Grans (9) | 2 – 67 | 932 | 694 (349) | 716 |
| Serial PBMC (10) | 2 – 67 | 1,074 | 808 (385) | 862 | ||
| CD4, CD8, CD20, CD14, CD18, CD34 | 63 – 64 | 632 | 477 (477) | 533 | ||
| Total | - | 1,527 | 1,120 (477) | 1,147 | ||
| RQ3570¶ | 160×106 (18%; 29×106 GFP+) | Serial Grans (6) | 2 – 38 | 2,213 | 1,809 (913) | 1,754 |
| Serial PBMC (7) | 2 – 38 | 3,965 | 3,295 (1,306) | 3,470 | ||
| CD4, CD8, CD20, CD14, CD18, CD34 | 36 – 37 | 2,664 | 2,095 (2,095) | 2,257 | ||
| Total | - | 5,758 | 4,586 (2,095) | 4,817 |
HSPC: hematopoietic stem cell and progenitor cell; EGFP: enhanced green fluorescent protein; QVI: quantifiable vector integrant; LT-QVI: long-term QVI; Genom. Sites: unambiguous genomic integration sites; PBC: total peripheral blood cells; Grans: granulocytes; PBMC: peripheral blood mononuclear cells.
Human immunodeficient virus (HIV)-based, self-inactivating lentivirus vectors (SIN-HIV vectors) expressing EGFP were used to transduce the CD34+ cells prior to transplant (An et al., 2001; Sander et al., 2006).
SIN-HIV vectors expressing the mutant viral herpes simplex virus-1 thymidine kinase (HSV1-sr39tk) gene were also used for animal 2RC003 for position emission tomography (Sander et al., 2006).
Simian immunodeficient virus (SIV)- based lentivirus vectors expressing both EGFP and shRNA against the HIV-1 co-receptor, C-C chemokine receptor type 5 (CCR5), were used to transduce the CD34+ cells prior to transplant (An et al., 2007).
The number of infused CD34+ HSPCs is shown. The levels of EGFP marking at infusion and the estimated number of EGFP-marked cells are shown within parenthesis.
The cell types analyzed in this study. For longitudinal analysis, PBCs or Grans/PBMCs were analyzed (the number of time points is indicated in the parenthesis). CD4+ and CD8+, CD20+, CD14, CD18+, and CD34+ cells were also analyzed near the experimental endpoints.
The total number of unique vector integrants (Uniq. VI) per animal
The total number of QVI per animal. The total number of LT-QVI is provided in the parenthesis.
The total number unambiguously mapped genomic VIS loci (Genom.Site) per animal.