FIGURE 2.

Biodistribution and blood clearance of the AAV9NGA mutant in mice. BALB/c mice (n = 4) treated with AAV9 (gray bars) or AAV9NGA (white columns) were sacrificed 3 days (A) or 3 weeks (B) after administration to harvest different organs. Total genomic DNA was then extracted from lysates obtained from homogenized tissue samples. Viral genome copy numbers were normalized to the total number of copies of the mouse lamin gene in each sample as determined by quantitative PCR. For pharmacokinetic analysis (C), BALB/c mice (n = 4) were injected intravenously with 1 × 10¹¹ vg copies of AAV9 (solid circles) or AAV9/NGA (triangles) vectors through the tail vein. Viral genome copy numbers were determined by qPCR from 10-μl aliquots of whole blood collected by nicking the tail vein at different time intervals. Error bars represent mean ± S.E. ***, p < 0.005.