AAV9/GFP, but not AAV9/GAN, vectors show a transient liver toxicity and reduction in vector genomes. Mice were injected with either ssAAV9/CBA-GFP (n = 6) or ssAAV9/CBA-GAN (n = 8). (a) Just after the 4-week time point postinjection, a group of mice (n = 3 green fluorescent protein (GFP), n = 4 giant axonal neuropathy (GAN)) was killed to assess biodistribution by quantitative PCR. In panels b–e, the arrow denotes the time when these mice were removed from the cohorts. (b) Weights were taken at the indicated time points and expressed relative to the starting weight. Serum was collected at the indicated time points and tested for levels of (c) albumin, (d) blood urea nitrogen (BUN), and (e) alanine amino transferase (ALT). All error bars indicate standard deviation. *Significantly higher value with P < 0.05 (Student's t-test: two-tailed, unpaired, equal variance). AAV9, adeno-associated virus serotype 9.