Supplementary Materials

This PDF file includes:

  • Fig. S1. GFP protein is detected in the spinal cord at the lumbar section.
  • Fig. S2. GFP protein is detected in the brain.
  • Fig. S3. GFP protein is detected in the pons and midbrain.
  • Fig. S4. Cellular immune response to GFP protein is quantified by the IFN-γ–ELISpot assay.
  • Fig. S5. Hepatic histopathology scores correlate with Ki67 immunostaining.
  • Fig. S6. Stable expression of miR-122 in the liver.
  • Table S1. Experimental groups, first M. fascicularis study.
  • Table S2. Liver transaminases, first M. fascicularis study.
  • Table S3. Statistical analysis for Fig. 3.
  • Table S4. Experimental groups, second M. fascicularis study.
  • Table S5. NAb titers to AAVrh.10 in serum and CSF after intrathecal delivery.
  • Table S6. Detection of viral genome in laser-capture microdissected motor neurons.
  • Table S7. Statistical analysis for Fig. 5.
  • Table S8. List of potential off-target genes based on sequence identity.
  • Table S9. RIN values.
  • Table S10. List of primers/probes.

[Download PDF]