Human F.IX transgenes expressed in mice
Construct . | Mutation . | hF.IX activity, % . | CRM status . | No. inhibitor patients/total no. patients . |
|---|---|---|---|---|
| WT | No mutation | 100 | + | NA |
| ES | Early stop codon (Arg29 → stop) | < 1 | − | 7/50 |
| LS | Late stop codon (Arg338 → stop) | < 1 | − | 0/25 |
| cCH | Missense as Chapel Hill dogs (Gly381 → Glu) | < 1 | − | Data unavailable |
| MS | Missense (Arg180 → Trp) | < 1 | + | 0/31 |
Construct . | Mutation . | hF.IX activity, % . | CRM status . | No. inhibitor patients/total no. patients . |
|---|---|---|---|---|
| WT | No mutation | 100 | + | NA |
| ES | Early stop codon (Arg29 → stop) | < 1 | − | 7/50 |
| LS | Late stop codon (Arg338 → stop) | < 1 | − | 0/25 |
| cCH | Missense as Chapel Hill dogs (Gly381 → Glu) | < 1 | − | Data unavailable |
| MS | Missense (Arg180 → Trp) | < 1 | + | 0/31 |
The 5 constructs generated and analyzed are listed. Data on antigen and activity levels, and tendency to inhibitor formation, are extracted from the hemophilia B database,25 or published data on the canine mutation.
NA indicated not applicable.