Supplementary Materials

The PDF file includes:

  • Materials and Methods
  • Fig. S1. Validation of LC-MS/MS methods and cellular experimental models.
  • Fig. S2. PK of ETV:IDS in wild-type mice.
  • Fig. S3. GAG reduction in fluids and peripheral tissue biodistribution of ETV:IDS in IDS KO mice.
  • Fig. S4. PK of ETV:IDS in TfRmu/hu mice.
  • Fig. S5. IDS deletion does not affect TfR levels in the brain.
  • Fig. S6. Individual disaccharide analysis from the brain and CSF of dosed IDS KO; TfRmu/hu KI mice.
  • Fig. S7. Characterization of FACS-isolated CNS cell types.
  • Fig. S8. Analysis of ganglioside, BMP, and glucosylceramide levels in FACS-isolated CNS cell types after treatment with ETV:IDS.
  • Fig. S9. Relationship between dose level and brain concentration of ETV:IDS.
  • Fig. S10. Analysis of lysosomal lipids in CSF after chronic treatment with ETV:IDS.
  • Table S1. LC-MS acquisition parameters information for the BMP and gangliosides assay.
  • Table S2. LC-MS acquisition parameters information for GlcCer and GalCer assay.
  • Table S3. LC-MS acquisition parameters information for eicosanoid assay.
  • Table S4. LC-MS acquisition parameters for lipidomics assay in negative ionization mode.
  • Table S5. LC-MS acquisition parameters for lipidomics assay in positive ionization mode.
  • Table S6. Targeted lipidomic analysis of IDS KO; TfRmu/hu KI mice after repeated administration of ETV:IDS or idursulfase.
  • Legend for data file S1
  • References (7588)

[Download PDF]

Other Supplementary Material for this manuscript includes the following: