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. Author manuscript; available in PMC: 2021 Jun 3.
Published in final edited form as: Mol Genet Metab. 2020 Sep 30;131(3):306–315. doi: 10.1016/j.ymgme.2020.09.005

Fig. 4.

Fig. 4.

Liver PAH Fluorescent Immunohistochemistry of Gene Therapy Treated PahΔexon1/Δexon1 Mice. Anti-PAH fluorescent-immunohistochemistry in liver tissue samples from gene addition therapy treated PahΔexon1/Δexon1 mice. Green indicates the presence of PAH by anti-PAH antibodies bound by secondary antibodies conjugated with AlexaFluor 488 (green fluorescence) and blue indicates nuclear staining with DAPI. The four images display the gene therapy treated PahΔexon1/Δexon1 mice as labelled according to dosing of therapeutic vectors of LSPmPAH AAV2/8: 1 × 1012 vg (top left image labelled with 1E12), 1 × 1011 vg (top right image labelled with 1E11), and 1 × 1010 vg (bottom left image labelled with 1E10), followed by 1 × 1012 vg of the control vector (LSPmPAHfs) containing a frameshift (bottom right image labelled with 1e12fs).