Fig. 2. NTBC-independent weight gain and correction of biochemical parameters after in vivo LV-FAH delivery.
a Timeline of NTBC cycling and withdrawal for No. 166 and No. 167 (first row) demonstrated NTBC-independent growth at 98 and 78 days post-treatment. Each gray bar represents 1 week spent on NTBC. Green line represents when blood was drawn for the biochemical data in (b). Timeline of NTBC undertreatment for No. 266, which did not receive LV-FAH and developed HCC, and for No. 158, which did not receive LV-FAH but received appropriate NTBC cycling. b Normalization of tyrosine, aspartate aminotransferase (AST), alkaline phosphatase (ALP), ammonia, and total bilirubin levels at 142 days post-treatment. Mean ± standard deviation are shown for No. 166 and No. 167 alongside historical wild-type (n = 2) and untreated FAH−/− (n = 3) controls. P-values are provided for treated animals compared to untreated FAH−/− controls based on a two-sided Welch’s t-test with no adjustment for multiple comparisons: * represents P < 0.05, ** represents P < 0.01, **** represents P < 0.0001. Tyrosine: p < 0.0001; AST: p = 0.01; ALP: p = 0.004; ammonia: p = 0.03; bilirubin: p = 0.04. c Age-appropriate decreases and maintenance of minimal alpha-fetoprotein (AFP) levels in No. 166 and No. 167 despite chronic NTBC withdrawal.
