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analysis of the results on CIS formation in clinical samples derived from 2 clinical X-SCID gene therapy studies [unpublished data].

<p>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 2^nd order was 30 kb. CIS, number of identified CIS of 2^nd 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.