Table 1.

Serological Measures of Functional Correction

Mice treatment	a Fah−/− off NTBC	b Fah−/− on NTBC	c Wild-type	d Fah−/− Van	e Fah−/− AAV	f Fah−/− AAV + Van	g sTx recipients AAV + Van
Bili (mg/dl)	3.4±1.3	0.2±0	0.0±< 0.1	<0.1±0	<0.1±0	<0.1±0	0.2±0.1
AST (U/L)	353±86	121±47	91±27	98±26	86±8	91±32	116±13
ALT (U/L)	291±51	59±56	36±5	38±4	45±1	53±12	92±10
n	6	5	5	3	3	7	6

AAV, adeno-associated virus; AST, aspartate aminotransferase; ALT, alanine aminotransferase; Bili, bilirubin; Fah, fumarylacetoacetate hydrolase; n, number of animals; NTBC, 2-(2-nitro-4-trifluoromethylbenzoyl-1,3-cyclohexanedione; sTx, serial transplant; U/L, units per liter; Van, vanillin. From left to right, values represent mean±SD from untreated Fah^−/− mice off NTBC, untreated Fah^−/− mice on NTBC, untreated wild-type mice, Fah^−/− mice treated with vanillin alone for 7 days on NTBC, Fah^−/− mice treated with AAV alone and selected for 3 months off NTBC, Fah^−/− mice treated with AAV and vanillin and selected for 3 months off NTBC, and serially transplanted Fah^−/− mice selected for 3 months off NTBC, respectively. All values in columns (b–g) are significantly different from corresponding values in column (a) (p<0.05).