Figure 1.

Clearance of self-complementary AAV vector (scAAV)2/8-LP1-hFIXco vector after peripheral vein administration of 2 × 10¹² pcr-vector genomes (vg)/kg. Clearance of the vector from rhesus plasma, urine, saliva, and stool was determined using a quantitative PCR (qPCR) assay on samples collected following peripheral vein administration of 2 × 10¹² pcr-vg/kg scAAV2/8-LP1-hFIXco. Standards consisting of serial dilutions of scAAV2/8-LP1-hFIXco in rhesus plasma were used to define the sensitivity of the assay. Results are expressed as mean transgene copy number (vg)/ml ± SE of sample obtained from three animals in this dose cohort and indicate that the vector genomes have cleared from the body fluids by day 10.