Table 1.
Plasmids and viral vectors used
| Plasmid or virus name | Promoter/Enhancer | cDNA |
|---|---|---|
| phCEFI-hAAT | hCEFI | Alpha-1 antitrypsin (hAAT) cDNA |
| phCEFI-sohAAT | hCEFI | Codon-optimised CpG-depleted human alpha-1 antitrypsin (hAAT) cDNA |
| pCIK-hAAT | CMV | Alpha-1 antitrypsin (hAAT) cDNA |
| pCIK-sohAAT | CMV | Codon-optimised CpG-depleted human alpha-1 antitrypsin (hAAT) cDNA |
| pCIK-Lux | CMV | Firefly luciferase (Lux) cDNA |
| pCIK-GLux | CMV | Gaussia luciferase (Glux) cDNA |
| rSIV.F/HN-hCEF-soGlux | hCEF | Codon-optimised CpG-depleted GLux cDNA |
| rSIV.F/HN-hCEF-sohAAT | hCEF | Codon-optimised CpG-depleted hAAT cDNA |
| r.SIV.F/HN-CMV-FVIII-N6 | CMV | codon-optimised FVIII containing 226 amino acid residues of the B domain |
Note: for pragmatic reasons (availability), the CMV rather than the hCEF promoter/enhancer were used for all FVIII studies. The hCEF promoter is comprised of a CpG-free form of the human CMV immediately/early enhancer and a CpG-free form of the human Elongation Factor 1a promoter. Plasmids (phCEFI-hAAT and phCEFI-sohAAT also contain a synthetic recombinant intron