Fig. 4: Correction of fatal liver disease with in vivo delivery of TnpB-ωRNA via single AAV. | Nature Communications

Fig. 4: Correction of fatal liver disease with in vivo delivery of TnpB-ωRNA via single AAV.

From: Engineering a transposon-associated TnpB-ωRNA system for efficient gene editing and phenotypic correction of a tyrosinaemia mouse model

Fig. 4

a Diagram of AAV-TnpB-ωRNA/ωRNA* vector and gene therapy schematics in Fah−/− mouse model of type I hereditary tyrosinaemia. b Survival curve for disease mice treated with AAV-TnpB-ωRNA/ωRNA* or AAV-TnpB without ωRNA (TnpB only group). c Body weight change during the observation period for disease mice in different treatment groups. d Body weight ratio for TnpB-ωRNA or TnpB only versus TnpB-ωRNA*-treated mice in 19-day after NTBC withdrawal. e Histology analysis with H&E and Sirius red staining for mouse liver from different treatment groups. f Western blot for HPD protein from untreated and TnpB-ωRNA-treated HT1 mice. Data are represented as means ± SEM. A dot represents a biological replicate (n = 3 or more). Unpaired two-tailed Student’s t tests. Significant differences between conditions are indicated by an asterisk (*P < 0.05, **P < 0.01, ***P < 0.001, ****P < 0.0001, NS non-significant.). Scale bars, 200 μm. Source data are provided as a Source Data file.

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