rAAV-Mediated Globin Gene Transfer Into Erythroleukemia (K562) Cells
. | MOI . | mRNA (RT-PCR) . | Integration (Southern blot)* . | |||
---|---|---|---|---|---|---|
. | . | Day 18† . | Day 35† . | Integrated† . | Unrearranged‡ . | Tandem Copiesρ . |
rHS432Aγ* | 3 × 108 | 12/12 | 19/23 | 9/16 | 5/9 | 0/5 |
rHS32Aγ*3′RE | 4 × 108 | 10/10 | ND | 8/18 | 8/8 | 6/8 |
rHS32Aγ*3′RE | 4 × 109 | 10/10 | ND | 23/24 | 23/23 | 22/23 |
. | MOI . | mRNA (RT-PCR) . | Integration (Southern blot)* . | |||
---|---|---|---|---|---|---|
. | . | Day 18† . | Day 35† . | Integrated† . | Unrearranged‡ . | Tandem Copiesρ . |
rHS432Aγ* | 3 × 108 | 12/12 | 19/23 | 9/16 | 5/9 | 0/5 |
rHS32Aγ*3′RE | 4 × 108 | 10/10 | ND | 8/18 | 8/8 | 6/8 |
rHS32Aγ*3′RE | 4 × 109 | 10/10 | ND | 23/24 | 23/23 | 22/23 |
Abbreviation: ND, not determined.
Southern blot analysis was performed on DNA extracted 35 to 50 days after transduction.
Ratio of positive clones to number of clones analyzed.
Proportion of clones that contain an unrearranged rAAV genome.
ρ Proportion of clones in which the integrated, intact genome was in a tandem array of multiple copies.