Table 1

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.

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