TABLE 2.
Design of AAV‐PAH mouse study. WT and Pahenu2 mice were assigned to one of six treatment groups with escalating doses of two variants of Anc80‐coPAH, an AAV vector expressing codon optimized PAH either with or without an internal intron under the control of the liver‐specific albumin enhancer and A1AT promoters. Mice were kept on study for 4 or 12 weeks after a single administration of vector on day 1. Mice underwent a full necropsy at the end of the study to demonstrate transduction and expression of the vector‐mediated PAH transgene, as well as routine toxicological evaluation
| Group | Genotype | Article | Dose (VG/kg) | Day 29 (M/F) | Day 85 (M/F) |
|---|---|---|---|---|---|
| 1 | Pahenu2 | Saline | Saline | — | 3/3 |
| 2 | WT | Saline | Saline | — | 3/3 |
| 3 | Pahenu2 | Anc80‐coPAH | 5 × 1011 | 3/3 | 2/2 |
| 4 | Pahenu2 | Anc80‐coPAH | 5 × 1012 | 3/3 | 2/2 |
| 5 | Pahenu2 | Anc80‐int‐coPAH | 5 × 1011 | 3/3 | 2/2 |
| 6 | Pahenu2 | Anc80‐int‐coPAH | 5 × 1012 | 3/3 | 2/2 |
Abbreviations: A1AT, alpha‐1 antitrypsin; AAV, adeno‐associated virus; PAH, phenylalanine hydroxylase gene.