Targeting of AAV capsid mutants selected on murine lung tissue <i>in vivo.</i>

<p>AAV luciferase vectors displaying selected or control capsids (wild-type or random insert VRRPRFW) were injected intravenously into female FVB mice. Tissue was harvested after 8 or 28 d, respectively, and processed as indicated. A: Evaluation of lung homing. Lung tissue was harvested 8 days after vector injection and the amount of AAV genomes was determined by quantitative PCR. Data represent mean values from n = 3 mice per group, analyzed in triplicates ± SD. B: <i>In vivo</i> lung gene transfer by selected AAV after intravenous injection. Lung tissue was harvested 28 days after vector injection, and luciferase activity was determined as relative light units (RLU) per mg protein. Bars indicate the median value, n = 5 mice per group (** = p<0.001 targeted vectors vs. wild-type and random insert control). C: <i>In vivo</i> transduction of various tissues in mice by AAV library mutants selected for lung transduction. Tissues were harvested and luciferase activity was determined as in 5B. The dotted line indicates the threshold beyond which luciferase expression data could be reliably delineated from background signal. Data represent mean values ± SEM, n = 5 mice per group. * p<0.05; ** p<0.01 targeted vectors vs. wild-type AAV-2. # p<0.05; ## p<0.01 targeted vectors vs. random insert control.