Figure 1

(A) Schematic of murine micro-dystrophin construct. A truncated MCK promoter/enhancer (563 bp) is used to drive muscle specific gene expression. Also labeled is a chimeric SV40 intron (97 bp) and synthetic polyadenylation site (53 bp). The 3,590 bp murine micro-dystrophin construction is depicted in detail. ABD is the complete actin binding domain, hinges 1, 2 and 4 are shown (green boxes), as are the flanking spectrin rod domains (SR blue boxes). The cysteine-rich dystroglycan binding domain is denoted by an orange box. AAV2 ITR are shown as arrowheads. (B) Immunofluorescence detection of micro-dystrophin expression in mdx mouse TA muscle. rAAV1, 6, or 8.micro-dystrophin (10¹¹ vg) was delivered by intramuscular injection (IM Control) or ILP through the femoral artery of 3–4 week old mdx mice. Representative TA muscle sections (12 um) are shown from 4 week post-injected animals (8 and 12 weeks looked similar). Sections were immuno-stained with the N-terminal dystrophin antibody Manex1a. Scale Bar, 50 μm.