Fig. 4
Oral administration of VCE-003.2 is neuroprotective from mutant-huntingtin-induced neurodegeneration. Wild type mice were injected bilaterally with mutant huntingtin expressing AAV-htt94Q (mtHtt) and AAV-htt16Q (wtHtt) as control. VCE-003.2 or vehicle were administered orally daily (10 mg/kg) and mice analyzed 30 days after lesion. Confocal microscopy characterization of huntingtin-CFP and using an antibody for the MSN marker DARPP32. Quantification of MSN survival after lesion for the indicated experimental groups. AAV-htt16Q (Vehicle and VCE-003.2, n = 5 each) and AAV-htt94Q (Vehicle and VCE-003.2, n = 7 each). Statistics: One-way ANOVA. a F = 31.56; R2 = 0.80. ##p < 0.01; q = 5.28 AAV-htt94Q VCE-003.2 vs. AAV-htt94Q Veh. **p < 0.01; q = 10.63 vs AAV-htt94Q Veh vs. AAV-htt94Q Veh vs. AAV-htt16Q Veh and **p < 0.01; q = 5.56 vs AAV-htt94Q VCE-003.2 vs. AAV-htt16Q Veh. b F = 27.15; R2 = 0.78. ##p < 0.01; q = 10.74 AAV-htt94Q VCE-003.2 vs. AAV-htt94Q Veh and **p < 0.01; q = 10.85 vs AAV-htt94Q Veh vs. AAV-htt16Q Veh. Scale bar, 100 μm