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. 2020 May 13;17:1139–1154. doi: 10.1016/j.omtm.2020.05.004

Figure 4.

Figure 4

In vivo functional test of AAV8 variants on non-engrafted FRG mice.

(A) Representative immunohistochemical analysis of a naive FRG mouse liver transduced with AAV8, AAV8-E533K, and AAV8-RQNR variants encoding the ssAAV-LSP1-GFP-WPRE-BGHpA construct (1 × 1010 vg/mouse). Red, glutamine synthetase; green, vector-encoded GFP; blue, DAPI (nuclei). Scale bar, 100 μm. (B) NGS read distribution of AAV8 variants expressed as a percentage of total mapped reads, in the pre-injection AAV mix and in the DNA recovered from murine liver cells. (C) Representative immunohistochemical analysis of the liver from a naive FRG mouse transduced with AAV8, AAV8-E533K-N499D, and AAV8-RQNR-N499D variants encoding a scAAV-CAG-GFP-SV40pA construct. To enable comparison of the AAV8 transduction pattern, AAV8 was injected at 5 × 1010 vg/mouse (high dose) and 5 × 109 vg/mouse (low dose). Red, glutamine synthetase; green, vector-encoded GFP; blue, DAPI (nuclei). Scale bar, 100 μm.