Figure 7.
In vivo targeting of dSaCas9-repressors to DUX4 exon 1 represses DUX4-fl and DUX4-FL targets in ACTA1-MCM;FLExD bi-transgenic mice
(A–F) dSaCas9-TRD or -KRAB ± sgRNAs were delivered intramuscularly using AAV9 to the ACTA1-MCM;FLExD moderate pathology FSHD-like transgenic mouse model, which carries one human D4Z4 repeat.70 Expression of DUX4-fl and DUX4-FL downstream markers Wfdc3 and Slc34a2 were assessed by qRT-PCR and normalized to levels of Rpl37. Copy-number ratios of dSaCas9-TRD or -KRAB to sgRNA are indicated. ∗p < 0.05, ∗∗p < 0.01 are from comparing to dSaCas9-TRD or -KRAB control.