Restoration of miR-185 expression eliminates TKI nonresponder cells regenerated in transplanted nonobese diabetic-Rag1^−/−IL2Rγc^−/− mice. (A) Detection of human GFP⁺CD45⁺ cells in the BM of mice injected with CD34⁺ CML patient cells transduced with a pRRL control vector (light blue), a miR-185 vector (light red), pRRL plus DA treatment (dark blue), or miR-185 plus DA treatment (dark red) at weeks 4, 12, and 25 posttransplantation. (B) qRT-PCR results for BCR-ABL1 transcript levels relative to GAPDH in total BM cells of all mice analyzed at 25 weeks. (C) Representative FACS plots of human (CD45⁺) and GFP⁺CD45⁺ cells detected in the BM of mice analyzed at 25 weeks posttransplantation (left) and a summary of the levels of transduced GFP⁺CD45⁺ (solid bars) versus non-transduced GFP^-CD45⁺ (white bars, right) human cells in all mice studied. (D) Levels of human GFP⁺CD34⁺, CD34⁺CD38⁻, and myeloid cells detected in the BM of all mice analyzed at week 25 posttransplantation. (E) Representative FACS plots of human GFP⁺CD34⁺ cells in the BM of mice at week 25 posttransplantation. (F) Colony output of FACS-sorted human GFP⁺CD45⁺ cells harvested from the BM of mice 25 weeks posttransplantation of cells from a CML patient sample. Data shown, except where otherwise indicated, are the mean ± SEM of measurements from 2 cohorts of mice injected with transduced cells from 2 independent TKI nonresponder patients. Each dot/triangle represents 1 mouse. P values were calculated using a 2-tailed unpaired Student t test. No. = number.