Formulas based statistical analysis of the results on CIS formation in clinical samples derived from 2 clinical X-SCID gene therapy studies [unpublished data].

Calculations were performed on the haploid size of the human genome (3.12 × 106 kb) and on the basis of an IS skewing (25% of all IS) to the +/− 5 kb TSS region, for which an (*) uniform or a (§) triangular IS distribution, respectively, was assumed. 75% of IS were assumed to be uniformly distributed over the remaining human genome. The genomic window size chosen for CIS of 2nd order was 30 kb. 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.