TABLE 2.
Summary of the performance of chimeric AAV vectors in muscle and liver
| Liver | Muscle | |||
|---|---|---|---|---|
| a1at | hFIX | a1at | hFIX | |
| AAV1 | 1 | 1 | 4.6-6.3 | 5.4-8.9 |
| AAV12_9:1 | 1.6-10.3 | 1.4-5.4 | 2-3.4 | 1.8-3.1 |
| AAV12_1:1 | 3.8-17.4 | 6.6-23.7 | 8.5-11.7 | 2.3-7.1 |
| AAV12_1:9 | 2.4-13.5 | 5.3-22.7 | 1.4-2.9 | 1.6-3.0 |
| AAV2 | 2.1-5.7 | 2.7-12.2 | 1 | 1 |
Data from two parallel experiments using either human factor IX (hFIX) or human α1-antitrypsin (a1at) as reporter genes, levels of which were assayed by ELISA, are presented. Approximately 5 × 1010 particles were injected per mouse. For studies targeting liver, vectors were injected intravenously into C57BL6 mice. For muscle studies, factor IX vectors were administered to CD4 KO mice while a1at vectors were injected intramuscularly into C57BL6 mice. The range of expression in fold compared to that of AAV1 in liver and AAV2 in muscle at week 6 postinjection is shown. The basal expression levels of transgene expression for human factor IX and human α1-antitrypsin were designated “1”. The following vectors were used: CsCl-purified AAV1, AAV2, and chimeric AAV1 and AAV2 at ratios of 9:1, 1:1, and 1:9.