Table 1.
Summary of PCR genotyping of ZFN-stimulated gene targeting
| A1ATD-iPSC line | Clones analyzed |
Het.a | Homo. / Hemi. b |
Het. + additonal integrations c |
Homo. / Hemi. + additonal integrations c |
Non-targeted d |
|---|---|---|---|---|---|---|
| A | 84 | 45 | 3 | 23 | 8 | 5 |
| B | 18 | 10 | 2 | 3 | 3 | 0 |
| C e | 216 | 112 | 9 | 52 | 21 | 22 |
|
| ||||||
| Mean frequency [%] | 54 | 6 | 23 | 12 | 5 | |
a
Het., clones heterozygous for PB allele.
b
Homo./Hemi., clones homozygous or hemizygous for PB allele. Cells with one targeted allele and deletion of the other allele are undistinguishable from correctly targeted homozygous clones by PCR. Such cells are designated as hemizygotes.
c
Vector backbone integration was analyzed by PCR.
d
Clones showing incorrect PCR bands are included.
e
A sum of 2 independent experiments.