Components of AAV vectors for expression of muscle-derived human F.IX (hF.IX)
| Vector . | Enhancer/Promoter . | Intron . | Human F.IX 3′-UT . | PolyA Signal . |
|---|---|---|---|---|
| AAV-CMV-hF.IX-3 | CMV IE | Portion of hF.IX intron I (1.4 kb) | 0.3 kb | SV40 late |
| AAV-CMV-hF.IX-3/bGH | CMV IE | Portion of hF.IX intron I (1.4 kb) | 0.3 kb | Bovine growth hormone (bGH) |
| AAV-HSA-hF.IX-1 | Human α-skeletal actin (HSA) | Portion of hF.IX intron I (0.3 kb) | 0.3 kb | SV40 late |
| AAV-CMV-hF.IX-8 | CMV IE | Portion of hF.IX intron I (0.3 kb) | Entire 1.6 kb | Human F.IX |
| AAV-CMV-hF.IX-2 | CMV IE | Chimeric CMV/β-globin mini-intron | 1.1 kb | Human growth hormone (hGH) |
| AAV-HSA/CMV-hF.IX-2 | HSA/CMV IE | Chimeric CMV/β-globin mini-intron | None | hGH |
| AAV-HSA-hF.IX-3 | HSA | HSA intron I | None | hGH |
| AAV-cTnT-hF.IX | Cardiac troponin I | Chimeric CMV/β-globin mini-intron | 1.1-kb | hGH |
| Vector . | Enhancer/Promoter . | Intron . | Human F.IX 3′-UT . | PolyA Signal . |
|---|---|---|---|---|
| AAV-CMV-hF.IX-3 | CMV IE | Portion of hF.IX intron I (1.4 kb) | 0.3 kb | SV40 late |
| AAV-CMV-hF.IX-3/bGH | CMV IE | Portion of hF.IX intron I (1.4 kb) | 0.3 kb | Bovine growth hormone (bGH) |
| AAV-HSA-hF.IX-1 | Human α-skeletal actin (HSA) | Portion of hF.IX intron I (0.3 kb) | 0.3 kb | SV40 late |
| AAV-CMV-hF.IX-8 | CMV IE | Portion of hF.IX intron I (0.3 kb) | Entire 1.6 kb | Human F.IX |
| AAV-CMV-hF.IX-2 | CMV IE | Chimeric CMV/β-globin mini-intron | 1.1 kb | Human growth hormone (hGH) |
| AAV-HSA/CMV-hF.IX-2 | HSA/CMV IE | Chimeric CMV/β-globin mini-intron | None | hGH |
| AAV-HSA-hF.IX-3 | HSA | HSA intron I | None | hGH |
| AAV-cTnT-hF.IX | Cardiac troponin I | Chimeric CMV/β-globin mini-intron | 1.1-kb | hGH |
Vector genomes were designed to approximate the size of wild-type AAV (approximately 4.5 kb) in order not to exceed the packaging limit for generation of recombinant AAV. All vectors include the entire coding region of the human F.IX cDNA plus the components listed above.