Research Article

An Intrabody Drug (rAAV6-INT41) Reduces the Binding of N-Terminal Huntingtin Fragment(s) to DNA to Basal Levels in PC12 Cells and Delays Cognitive Loss in the R6/2 Animal Model

Figure 1

INT41 reduces Htt-exon1-GFP (Q103) aggregation in HEK293T cells. HEK293T cells were transfected with vectors encoding Htt-exon1-GFP with either a short (Q30) or extended (Q103) glutamine repeat region. Vectors encoding the intrabodies Happ1 or INT41 were cotransfected with Htt-exon1-GFP (Q103). After 40 hours, flow cytometry analysis on a FACS Aria was performed and GFP signals were gated into three categories based on GFP intensity. Htt aggregates are represented in the >100 signal category. The percentage of cells in each category for each treatment is shown.