In vitro and in silico characterization of competitive inhibition and repression of DUX4 target gene activation as a therapeutic approach for facioscapulohumeral muscular dystrophy (FSHD)
It is shown that DBD alone produces dose-dependent repression of DUX4-FL transcriptional activity in HEK293T cells, while a constitutively expressed DBD-KRAB fusion produces significantly greater repression than DBD alone, with a similar trend observed in C2C12 myoblasts.
H. Hoffmann, A. Finkelstein, Amanuel W. Geremew et al.
· bioRxiv · 0 citations