MiR-139-3p, miR-199a-3p, and their targets are aberrantly expressed in FA. (A) Luciferase reporter assays containing the 3′-UTR sequences of indicated targets of miR-139-3p or miR-199a-3p with WT or mutated miRNA binding sites. Luciferase activities relative to the EV control are shown. Error bars represent the SD of 3 experiments. The significance was calculated with the Student t test (2-tailed) (*P < .05). (B) Expression of miR-139-3p and miR-199a-3p normalized to U6 in BM CD34⁺ cells isolated from healthy individuals (nBM) and FA patients. Data are plotted relative to the average of nBM. Patients with severe BMF are indicated with a circle. The bars show the median relative expression of each group. The significance was calculated with the Mann-Whitney U test (asymptotic significance [2-tailed], *P< .05). (C) Transcript levels of indicated miR-139-3p or miR-199a-3p targets, TP53, and ACTB in low-density mononuclear BM cells from 21 FA patients and 11 healthy volunteers (normal) are shown. Data were taken from the FA Transcriptome Consortium database (Gene Expression Omnibus, 2009 and publicly available as GSE16334). The bars indicate the mean relative expression of each group. The significance was calculated with the non-paired Student t test (asymptotic significance [2-tailed], *P < .05). nBM, normal BM.