Supplementary Materials

The PDF file includes:

  • Fig. S1. ASM colocalizes with the lysosomal marker Lamp1.
  • Fig. S2. AAV9-hASM CM injection does not promote immune response in brain tissue of NHP.
  • Fig. S3. Expression of hASM does not differ among cerebellar lobes in AAV9-hASM iCb- and CM-injected ASM-KO mice.
  • Fig. S4. Purkinje cell death is more evident in cerebellar lobes of AAV9-hASM iCb- compared to CM-injected ASM-KO mice.
  • Fig. S5. Astrocytosis and microgliosis are higher in cerebellar lobes of AAV9-hASM iCb- compared to CM-injected ASM-KO mice.
  • Fig. S6. AAV9-hASM iCb-injected ASM-KO mice show higher ceramide levels than AAV9-hASM iCM-injected ASM-KO mice.
  • Fig. S7. ASM-KO mice noninjected and CM injected with AAV9 empty vector or aCSF show similar behavioral, cellular, and molecular pathology.
  • Fig. S8. Statistical comparison of the effects of cerebellar iCb and CM injection in ASM-KO mice.
  • Fig. S9. CM injection of AAV9-hASM promotes hASM expression along the rostrocaudal axis.
  • Fig. S10. hASM mRNA is not detected in the liver of AAV9-hASM CM-injected ASM-KO mice by RT-PCR.
  • Fig. S11. ASM activity in plasma of aCSF-injected WT is higher than in ASM-KO AAV9-hASM CM-injected mice.
  • Fig. S12. CM injection results in a vast transduction of different components of the ventricular system and surrounding areas.
  • Fig. S13. AAV9-hASM infects primary cortical neurons of ASM-KO mice and elicits secretion of hASM.
  • Table S1. AAV9-hASM CM injection does not affect general health, appearance, and behavior of NHP as determined by monthly average of daily observations.
  • Legend for data file S1

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Other Supplementary Material for this manuscript includes the following: