FIG. 1.

Long-term survival of Mut^–/– mice after liver-directed gene therapy. Survival (in days) of untreated Mut^–/– mice (n = 58) and those treated with either 2 × 10¹¹ GC (n = 15) or 4 × 10¹¹ GC (n = 6) of rAAV8-TBG-mMut in the neonatal period. Mice treated with 4 × 10¹¹ GC of AAV8-TBG-mMut (n = 6) had 84% survival (5 of 6) by 360 days of life compared with 53% (8 of 15) for those treated with 2 × 10¹¹ GC of AAV8-TBG-mMut. Both treated groups had significantly increased survival at 24, 60, and 100 days and beyond (*p < 0.01) compared with the group of untreated Mut^–/– mice.