Table 2.
Cell line | Mutation GAA1/GAA2 | Reprogramming | No. of clones |
---|---|---|---|
68 | 570/1200 | OSKM Retro | 5 |
4491 | 559/G130V | OSKM Retro | 3 |
GM03665 | 816/1400 | OSKM Retro | 3 |
GM04078 | 341/480 | OSKM Retro | 4 |
68_E | 570/– and 1200/– | OSKM Retro | 2 |
68 | 570/1200 | OSKM Sendai | 3 |
4259 | 404/920 | OSKM Sendai | 2 |
281 | 630/806 | OSKM Sendai | 3 |
Lines designated GM were derived using fibroblasts obtained from Coriell Cell Repositories. Line 68_E is a zinc finger-edited line with one GAA allele corrected by excision of the expanded sequence.15 Two different methods, retroviral and Sendai virus transduction, were used to establish these lines. All lines underwent rigorous testing for pluripotency, lineage differentiation, and karyotype analyses. As GAA repeats continuously expand in FRDA iPSCs, the repeat number is based on determination in fibroblasts prior to reprogramming.