Fig. 6.

Improvement in lifespan following AAV-mediated gene therapy with or without phosphocysteamine. The median lifespan of the untreated Ppt1-/-mice was 36.5 weeks compared to 34 weeks in the phosphocysteamine-treated mutant mice. There was a significant improvement in lifespan in mutant mice treated with either AAV only or AAV+phosphocysteamine. Treatment with AAV only increased their median lifespan to 54 weeks while AAV+ phosphocysteamine increased median lifespan to 59 weeks, although this difference was not significant