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. 2018 Mar 1;29(3):299–311. doi: 10.1089/hum.2017.095

Figure 1.

Figure 1.

A schematic outline of the dual AAV ΔH2–R15 mini-dystrophin vectors. YZ37 (vector size = 4.7 kb) carries the 5′ part of the minigene and YZ39 (vector size = 4.9 kb) carries the 3′ part. A flag gene is fused in-frame to the N-terminal end of the mini-dystrophin gene and a GFP gene is fused in-frame to the C-terminal end. Homologous recombination between the overlapping region (indicated by dotted box) reconstitutes the complete 7-kb mini-dystrophin gene expression cassette (size = 8.6 kb) and leads to the production of a 250-kD mini-dystrophin protein). AAV, adeno-associated virus; C, C-terminal domain of dystrophin; CMV, cytomegalovirus promoter; CR, cysteine-rich domain of dystrophin; H, proline-rich hinges in the dystrophin rod domain; N, N-terminal domain of dystrophin; pA, polyadenylation signal; trapezoid structures, inverted terminal repeats of AAV. Numerical digits, spectrin-like repeats (R) in the dystrophin rod domain.