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In vivo analysis of functionality of rhLDLR-21 and rhLDLR. A: Western blot examining protein expression of rhLDLR and rhLDLR-21 at a dose of 1 × 1011 genome copies (GC)/mouse 21 days after intravenous tail vein injection. Plasma cholesterol and ALT levels (B) in Ldlr−/−Apobec1−/− mice after treatment with 1 × 1011 GC/mouse or 1 × 1010 GC/mouse AAV8.TBG.RhLDLR or AAV8.RhLDLR-21 (n=5 animals per dose group). Each point represents mean ± SD.
