Table 1.
Changes in DUX4-dependent gene expression following targeting of dSaCas9-repressors to DUX4
| dSaCas9-TRD | dSaCas9-SET | dSaCas9-HP1γ | dSaCas9-HP1α | |
|---|---|---|---|---|
| KREMEN2 | 1.33 | 1.57 | 1.64 | 1.26 |
| FRAS1 | 1.08 | 1.15 | 1.05 | NS |
| TRIM14 | 1.05 | 1.10 | 1.16 | 1.03 |
| FRZB | NS | NS | 1.02 | 1.09 |
| COL9A2 | NS | NS | 1.15 | NS |
| TYMP | 1.53 | 1.63 | 1.70 | 1.30 |
| CMPK2 | 4.47 | 4.80 | 4.82 | 4.12 |
| SPTBN5 | 1.14 | 1.13 | 1.23 | 1.02 |
| GRIA1 | 1.09 | 1.18 | 1.39 | 1.33 |
| TPPP3 | 1.07 | 1.28 | 1.20 | 1.01 |
| LY6E | 1.69 | 1.96 | 1.92 | 1.54 |
| PARP14 | 1.11 | 1.33 | 1.21 | 1.05 |
| ACSM5 | 1.13 | 1.45 | 1.35 | NS |
| PRELP | 1.01 | 1.02 | 1.05 | NS |
| TM6SF1 | −1.10 | −1.35 | −1.16 | −1.22 |
| ITGA8 | −1.24 | −1.36 | −1.37 | −1.19 |
| COL10A1 | NS | −1.02 | NS | −1.13 |
Shown are log2 fold changes in DUX4 target genes,61, 62, 63, 64, 65 whose expression in FSHD myocytes is altered following transduction with each dSaCas9-repressor + DUX4-targeting sgRNA. These genes are part of developmental pathways dysregulated by DUX4, and their differential expression following CRISPRi treatment represents a return to a more normal pattern of gene expression. NS, not significant.