RIS distribution in T-cell clones from ADA-SCID patients
. | Intragenic, % . | Intergenic, % . | Less than 10 kb upstream, % . | Within 5 kb from TSS, % . |
---|---|---|---|---|
T-cell clones | 29.3 | 70.7 | 29.3 | 36.6 |
Ex vivo follow-up | 41.3 | 58.7 | 19.6 | 28.8 |
Pretransplant CD34+ cells | 50.9 | 49.1 | 12.3 | 23.6 |
. | Intragenic, % . | Intergenic, % . | Less than 10 kb upstream, % . | Within 5 kb from TSS, % . |
---|---|---|---|---|
T-cell clones | 29.3 | 70.7 | 29.3 | 36.6 |
Ex vivo follow-up | 41.3 | 58.7 | 19.6 | 28.8 |
Pretransplant CD34+ cells | 50.9 | 49.1 | 12.3 | 23.6 |
Distribution of RV integration sites mapped in T-cell clones isolated ex vivo from the peripheral blood of patients 2 and 3 at 18 months after gene therapy, compared with RV integrations in pre-GT and post-GT bulk samples from a previously published collection.11 Integrations were distributed as inside (intragenic) or outside (intergenic) RefSeq genes, and at a distance of less than 10 kb upstream or plus or minus 5 kb from TSSs.