Body weight of MMA mice and their heterozygous littermates.

(A) Animals were dosed with vehicle or 1×1014 vg/kg mLB-001 on PND 1. Nursing dams were kept on the standard 21% protein diet until PND 14, when the diet was switched to a 12% protein diet. Pups were weaned on PND 28 and maintained on the 12% protein diet. At 3 months of age, all surviving animals were switched to the standard chow with 21% protein content and then to a 40% protein diet at 4 months of age. Animals were monitored until 6 months of age. (B) Animals were dosed with vehicle, 2.5×1013, 5×1013 or 1×1014 vg/kg mLB-001 on PND 1. Other procedures are the same as for (A). (C) Animals were maintained on the 12% protein diet and dosed with vehicle or 5×1013 vg/kg mLB-001 at 8 weeks of age. At 3 months post-dosing, the diet was switched to the standard chow with 21% protein content and then to a 40% protein diet at 4 months post-dosing. Animals were monitored until 8 months of age. (TIF)