Figure 2.

Survival was extended in ASS^−/− mice after prenatal injection with rAAV2/8-LSP1mASS. ASS^−/− pups were injected prenatally at E16 with 2.5 × 10¹¹ vg of rAAV2/8-LSP1mASS. At birth, litter sizes were reduced to four pups per dam. Vector was then readministered at D0, D14 and/or D28 (n = 3 to four pups per group). All pups, including untreated controls (n = 3), received daily doses of L-arginine. (a) The survival curve shows the days on which ASS^−/− pups were culled due to the development of clinical signs of illness. (b) Plasma ammonia and (c) liver ASS activity were measured at the time of culling. Error bars represent SEM, *P < 0.05 versus ASS^+/+ controls. ASS, argininosuccinate synthetase.