Fig. 1. Branaplam and risdiplam treatment of HD LCLs produced two major HTT alternative splice products.

a Schematic diagram showing the alternative HTT splice products upon drug treatment. b PCR from exon 49–50 showing the size of the splice products from a single experiment. c Branaplam and risdiplam dose response for each HTT splice product. d Quantification of splice products produced from mutant minigenes following transfection of HEK 293 T cells either treated with a vehicle control (DMSO) or 100 nM branaplam. Source data are provided as a Source Data file.