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TTR-RB15

(A). (B). pAAV2-TTR-RB15 (used as marker). scAAV2-TTR-RB15. 3’. 5’. TTR-RB15. TTR-RB15 monomer, 2490 nt. Not-digested. Dimer, 4856 nt. Parent, 3967 nt. Monomer, 2490 nt. 3’. TTR-RB15. 5’. Self-complementary TTR-RB15 dimer, 4856 nt. RT-PCR. (C). PCR. scRB15- mutant. scRB15-

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TTR-RB15

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  1. (A) (B) pAAV2-TTR-RB15 (used as marker) scAAV2-TTR-RB15 3’ 5’ TTR-RB15 TTR-RB15 monomer, 2490 nt Not-digested Dimer, 4856 nt Parent, 3967 nt Monomer, 2490 nt 3’ TTR-RB15 5’ Self-complementary TTR-RB15 dimer, 4856 nt RT-PCR (C) PCR scRB15- mutant scRB15- mutant scRB15 scRB15 HepG2 Parent HepG2 Parent days 3 3 1 3 1 3 1 1 3 3 1 3 1 3 1 1 RB15 (D) P=0.005 ns P=0.001 ApoB mRNA in HepG2 (% of inactive RB15-mutant) Parent -AAV2-TTR -RB15-mutant scAAV2-TTR -RB15-mutant scAAV2-TTR -RB15 Parent-AAV2 -TTR-RB15 Ad-RB15 -mutant Ad-RB15

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