Figure 4.

In vivo administration of scAAV2/8 in a macaque thymus results in thymocyte transduction. (a) Three-year-old cynomolgus macaques were injected intrathymically in the right lobe with 2 × 10¹³ vector genomes–containing scAAV2/8 particles in two or three points. Ten days following injection, thymi were removed under general anesthesia and four sections were made from each lobe, shown here in the schematic diagram and labeled as A to D. (b) Transduction efficiencies in the various sections were assessed by quantitative PCR of the enhanced green fluorescent protein (eGFP) transgene. All values were normalized to Epo and the mean of eGFP copies per genomic DNA ± SD are shown for samples from the two macaques. (c) Flow cytometry data showing the percentages of eGFP⁺ cells as a function of side scatter (SSC) in the section B and A from right (right panels) and left (left panels) lobes, respectively, from a representative macaque injected 10 days previously with the scAAV2/8 vector (top row). The phenotypes of eGFP⁺ thymocytes in comparison to the total thymocyte population are shown as a function of CD4 and CD8 expression. scAAV, self-complementary adeno-associated virus.