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Editing of the MECP2 gene in Rett patient-derived cells with AAVHSC15-226 editing vector.
| Rett cell line | Mutation | % Mutation frequency | Location | Domain | Cell type | Sex | % Editing |
|---|---|---|---|---|---|---|---|
| GM11273 | R106W | 2.79 | Exon 3 | MBD | Fibroblast | F | 9.1 |
| GM17538 | S134C | 0.44 | Exon 4 | MBD | B-LCL | M | 3.8 |
| GM17880 | T158M | 8.74 | Exon 4 | MBD | Fibroblast | F | 8.6 |
| R282X | R282X | 5.74 | Exon 4 | TRD | Fibroblast | F | 12.1 |
| GM11270 | R306C | 5.14 | Exon 4 | TRD | Fibroblast | F | 11.1 |
