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Determination of Vector Genome Copy Numbera
| Subject | DNA loaded (μg) | Vector genome copy no. (per μg DNA) |
|---|---|---|
| AAV-tx GSDIa | 1.00 | 13,249 |
| WT | 1.00 | 0 |
| GSDIa | 1.00 | 0 |
Abbreviations: AAV-tx GSDIa, AAV-treated GSDIa animal; GSDIa, untreated GSDIa animal; WT, wild type.
AAV vector-derived genomes were detectable by real-time PCR in liver 6 months after secondary dosing. One microgram of DNA isolated from the liver was analyzed by quantitative real-time PCR and copy numbers are reported.
