Phenotypic Abnormalities of HD Cells Are Reversed in the Corrected hiPSCs
(A) Assessment of neural rosette formation following neural induction of CAG33, CAG180, and isogenic HD-C#1 and HD-C#2 control hiPSCs. Staining with Nestin, a marker for neural stem cells, and ZO-1, a luminal neural rosette marker, reveal impaired rosette formation in the CAG180 HD line compared with the control CAG33 lines. Correction of the HD mutation in the HD-C#1 and HD-C#2 rescues the HD-related impairment in neural rosette formation. Results are representative of three independent experiments.
(B) TUNEL immunofluorescence images showing cell death in CAG180 and HD-C#1 neurons following growth factor withdrawal.
(C) Quantification of cell death as measured by TUNEL staining in CAG180 and isogenic control neurons following growth factor withdrawal. n = 3 independent biological replicates; values shown as mean ± SEM, and ns, no significance and ∗∗p < 0.01 for shown comparison, §p < 0.05 and §§§p < 0.001 relative to CAG180-GF withdrawal group, was determined by unpaired t test.