Table 3.
Detection of rAAV6 capsid protein in mPB-CD34+ HSPCs at 48 h post-genome-editing treatment
| Sample | HR% | NHEJ% | VP3 |
|---|---|---|---|
| Untreated | N/A | N/A | not detecteda |
| rAAV6 (lot #3) MOI 2,500 rep1 (donor 3) | 56.67 | 33.92 | not detecteda |
| rAAV6 (lot #3) MOI 2,500 rep2 (donor 3) | 58.76 | 33.62 | not detecteda |
| rAAV6 (log #1) MOI 1,250 rep1 (donor 5) | 41.10 | 28.63 | not detecteda |
| rAAV6 (log #1) MOI 1,250 rep2 (donor 5) | 40.90 | 30.79 | not detecteda |
| rAAV6 (log #1) MOI 2,500 rep1 (donor 5) | 41.07 | 29.28 | not detecteda |
| rAAV6 (log #1) MOI 2,500 rep2 (donor 5) | 45.09 | 22.94 | not detecteda |
| rAAV6 (log #1) MOI 5,000 rep1 (donor 5) | 41.39 | 29.46 | not detecteda |
| rAAV6 (log #1) MOI 5,000 rep2 (donor 5) | 45.25 | 22.81 | not detecteda |
| rAAV6 (lot #2) MOI 2,500 rep1 (donor 5) | 43.52 | 26.22 | not detecteda |
| rAAV6 (lot #2) MOI 2,500 rep2 (donor 5) | 44.86 | 30.11 | not detecteda |
| rAAV6 (lot #1) MOI 2,500 (donor 7) | 52.77 | 29.93 | not detecteda |
| rAAV6 (lot #1) MOI 2,500 (donor 8) | 46.94 | 34.94 | not detecteda |
rAAV6, RNP/electroporation/rAAV6; rep, replicate.
a
Levels were lower than the reliable detection threshold, which was equivalent to 2.00E4 vg/cell.