In vitro expression of BDD hFVIII from AAV vectors
| . | Antigen Assay . | Function Assay . | |
|---|---|---|---|
| ELISA . | APTT . | Coatest . | |
| Transfection | 5.6 ng/mL | 25% | 28 mu/mL |
| Transduction | 15 ng/mL | 40% | 72 mu/mL |
| . | Antigen Assay . | Function Assay . | |
|---|---|---|---|
| ELISA . | APTT . | Coatest . | |
| Transfection | 5.6 ng/mL | 25% | 28 mu/mL |
| Transduction | 15 ng/mL | 40% | 72 mu/mL |
1 × 106 293 cells were transduced with rAAV/DLZ6 or rAAV/DLZ8 (EGFP) at MOI = 10. Media was harvested at 24 hours for hFVIII assay. The media overlay 293/EGFP was used as control. UCRP served as the standard, which is equivalent to 200 ng/mL hFVIII antigen and 1000 mu/mL Coatest activity. APTT refers to the percent of normal FVIII activity. Results are expressed as the mean of 3 experiments, each performed in triplicate.