Figure 1.

Design and testing of the constructs of recombinant adeno-associated viral vectors expressing the shRNA against FKRP. A: Schematic of pAAV-U6-shFKRP vectors. The FKRP2 and FKRP5 expression cassettes in the dual cassette lie in a head-to-tail orientation. The EYFP-tagged mouse FRKP plasmid was driven by the CMV promoter. B: Knockdown of FKRP-EYFP protein synthesis shown by Western analysis of 293 cells cotransfected with pFKRP-EYFP and pAAV-U6-shFKRP plasmids. FKRP-EYFP fusion protein, with a molecular weight of about 100 kDa, was detected by anti-GFP antibody, which cross-reacts with EYFP. Actin was stained as a sample loading control. Samples were prepared in duplicate.