Figure 1.

Intravenous Administration of GNeo-Conjugated IDUA Shows Delivery Restricted to Somatic Tissues

MPSI mice (Idua^−/−) were treated intravenously with 5 mg/kg GNeo-IDUA, unconjugated IDUA, or PBS. Tissues were harvested 1 hr following treatment. (A) IDUA enzyme activity in whole brains. (B) IDUA enzyme activity in livers. (C) Non-reducing end heparan sulfate analysis by liquid chromatography/mass spectrometry in whole brains 48 hr after injection of enzyme. (D) Non-reducing end heparan sulfate analysis in livers. n = 2–3 mice per bar. Error bars indicate SEM.