Table 1.
Human G6PC genome distribution and mRNA expression in 12-week-old high dose rAAV-GPE-treated G6pc−/− mice
Tissue | Human G6PC Copy number/μg genomic DNA |
Human G6PC mRNA relative to RpI19 mRNA × 105 |
---|---|---|
Wild type tissues, no transgene (n = 6) | 8 ± 2 | 8 ± 1 |
Liver, −/−/rAAV8-GPE (n = 6) | 94,440 ± 7,624 (100) | 62740 ± 4445 (100) |
Kidney, −/−/rAAV8-GPE (n = 6) | 2,429 ± 626 (2.57) | 22 ± 5 (0.033) |
Intestine, −/−/rAAV8-GPE (n = 6) | 601 ± 124 (0.64) | 9 ± 1 |
Brain, −/−/rAAV8-GPE (n = 6) | 121 ± 23 (0.12) | 8 ± 1 |
Testis, −/−/rAAV8-GPE (n = 6) | 25 ± 8 (0.02) | 9 ± 1 |
Ovary, −/−/rAAV8-GPE (n = 6) | 7 ± 2 | 9 ± 1 |
Data are means ± SEM. The values of wild type tissues, which contain no transgene, were the mean ± SEM of liver, kidney, intestine, brain, testis, and ovary. Numbers in parentheses are % of liver value.