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Investigating the Efficacy of AAV2-TTR-RB15 Variants in HepG2 Cells

This study examines different constructs of AAV2-TTR-RB15, including scAAV2-TTR-RB15 variants, to evaluate their effectiveness as markers and their impact on ApoB mRNA levels in HepG2 cells. Specifically, we compare the results of the parent and mutant RB15 variants over a series of time points. Key findings highlight significant differences (p=0.001, p=0.005) in the expression between the constructs. The results suggest that modified AAV2-TTR-RB15 variants could have potential applications in gene therapy.

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Investigating the Efficacy of AAV2-TTR-RB15 Variants in HepG2 Cells

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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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