Fig. 1.

Schematic representations of HTT gene structures in the HD sheep, minipig and NHP models. Gene structures were derived from: OVT73 transgenic sheep [10]; TgHD (N548) transgenic minipig [43]; KI-HD-150Q minipig [53]; KI-HD-85Q minipig (Exemplar Genetics personal communication and D. Howland; this article); transgenic NHP models: uHTT-84Q and hHTT-73Q [67]; and the somatic NHP AAV-HTT-82/85Q and AAV-HTT-16/10Q (J. Mcbride; this article) [73]. Lentiviral and AAV DNA vector elements are depicted in open white squares. BGH, bovine growth hormone; UTR, untranslated region; GFP, green fluorescence protein; Ubi (promoter), ubiquitin; CAG (promoter), cytomegalovirus early enhancer element/promoter-first exon-first intron of chicken B-actin gene/splice acceptor of rabbit B-globin gene; SV40/CMV/U5, simian virus 40, cytomegalovirus, unique 5; RRE/cPPT, rev response element, central polypurine tract; LTR, long terminal repeat; FLAP, a 3-stranded DNA structure; WPRE, Woodchuck Hepatitis Virus Posttranscriptional Regulatory Element; IRES, internal ribosome entry site; ITR, inverted terminal repeat.