Figure 3.
In vivo validation of dual AAV strategies. (A) In vivo comparison of dual strategies after subretinal injections using AAV8 or Anc80_L65 relative to AAV8 CMV.lacZ (LacZ) control at a dose of 3E9 vg/eye. (B) mRNA expression data comparing reconstituted genomes from HB at different doses (low: 3E9 vg/eye; high: 1.5E10 vg/eye). The delta-CT presented is normalized to GAPDH, relative to CMV.lacZ (LacZ). (C) Protein expression levels of HB in vivo at different doses (low: 3E9 vg/eye; high: 1.5E10 vg/eye). In vivo data are represented as mean ± S.E.M. from 2 to 3 assays done in duplicate to quadruplicate from 4 to 6 eyes per group.