Comparative analysis of mean values and p-values obtained computationally (‘Simulation’) or mathematically (‘Formula’).

The results refer to the presence of CIS detected in 2 clinical X-SCID gene therapy studies [unpublished data]. Simulations were performed with 50000 runs on the haploid size of the human genome (3.12×106 kb). P-values estimated from simulations equal the proportion per 50000 runs in which the number of CIS was at least as high as the number observed in the trials. The genomic window size chosen for CIS of 2nd order was 30kb. CIS, number of identified CIS of 2nd order in patient and control samples pre- and post-transplant; IS, number of all unique identified integration sites in patient and control samples pre- and post-transplant; MV, mean value.