Figure 2.

Delivery of AAV8-SpCas9 and AAV8-sgRNA4-Donor to Cure HT1 Rabbits

(A) Schematic views of the AAV vectors of SpCas9 and sgRNA4-Donor. (B) Key time points of AAV delivery and NTBC withdrawal. (C and D) Kaplan-Meier body weights (C) and survival (D) curves of the wild-type, AAV-treated, and AAV-untreated HT1 rabbits. (E–G) Biochemical analysis of the serum from the AAV-treated and AAV-untreated HT1 rabbits for the comparison of liver damage markers, namely, aspartate aminotransferase (AST), alanine aminotransferase (ALT), total bilirubin (TBIL), and direct bilirubin (DBIL) (E); renal damage markers, namely, blood urea nitrogen (BUN) and creatinine (CREA) (F); and the main biomarker, namely, succinylacetone (SA) and tyrosine (Tyr) (G). Error bars: mean ± SD. Asterisk indicates statistical significance as follows: ns, not significant; ∗p < 0.05; ∗∗p < 0.01; ∗∗∗∗p < 0.0001.