Fig. 1

Zinc finger (ZF) mouse host-adaptation design and rAAV2/1 transfer into the striatum. a Comparison of the ZF-KOX1 and mZF-KRAB zinc finger repressor designs, showing the 11-finger constructs aligned to their target poly(CAG) DNA sequence (mut HTT). Protein domains containing non-mouse peptide sequences (containing potential foreign epitopes) are shaded in red. The sequences of representative DNA recognition helices from fingers 2 and 3 (F2, F3) are displayed below the ZF arrays, with foreign sequences in red font. The percentage totals of non-mouse residues within the full length peptide sequences are given to show that the mouse-adapted design reduces overall non-host sequences. Full annotated sequences are provided in Additional file 1. b Representative specimen showing GFP expression in mouse coronal slices of hemi-brains, from anterior to posterior view. The anteroposterior (AP) location of each slice within the brain of the mouse is shown as AP distance from Bregma, following [39]. c Bar chart showing the average volume (±S.E.M) of the whole dorsal striatum and the volume covered by GFP fluorescence. Abbreviations: NLS, nuclear localization signal; ac, anterior commissure; cc, corpus callosum; DSt, dorsal striatum; LV, lateral ventricle. Scale bar: 1 mm