Repair of the ACTL6B c.1279del Mutation Restores Morphological and Dendritic Deficits
(A) Schematic detailing ACTL6B CRISPR repair.
(B) Sanger sequencing traces of a successful repair (SR) and unrepaired (UR) cell line generated from ACTL6Bext∗33 cell line.
(C) ACTL6B expression in SR and UR NPCs at a D5 time point (n = 6).
(D) Representative TUJ1 and MAP2 staining taken from SR and UR forebrain immature neurons at D15. Scale bars represent 40 μm.
(E) Quantification of the surface area of the nucleus and soma and the length of projections in the cell lines shown in (D) (n > 50).
Student’s t test, ∗p < 0.05, ∗∗p < 0.01, ∗∗∗p < 0.001.