Time course of rAAV2/8-HMBS-mediated hepatic HMB-synthase expression. AIP mice were sacrificed 1, 2, 6, 12, 24, and 36 weeks after vector administration and the hepatic HMB-synthase activities were determined. Results are shown as mean ± SD. Of note, the HMB-synthase activity at week 0 represents baseline activity in the AIP mice. The area between the broken lines represents the range of hepatic HMB-synthase activity detected in wild-type mice (n = 5). AIP, acute intermittent porphyria.