Science Advances

Supplementary Materials

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  • fig. S1. Designing sgRNA to target the human SOD1 gene.
  • fig. S2. CRISPR-Cas9 reduced mutant SOD1 expression in NSC-34–G93A–SOD1 cells by genome editing.
  • fig. S3. Quality control of AAV vectors.
  • fig. S4. Mutant SOD1 expression in the spinal cord of untreated G93A-SOD1 mice.
  • fig. S5. Systemic administration of AAV9-SaCas9-hSOD1 to neonatal G93A-SOD1 mice leads to SaCas9 expression in ChAT+ cells in the spinal cord.
  • fig. S6. Systemic administration of AAV9-SaCas9-hSOD1 to neonatal G93A-SOD1 mice leads to SaCas9 expression in β3-tubulin+ fibers in the spinal cord.
  • fig. S7. Systemic administration of AAV9-SaCas9-hSOD1 to neonatal G93A-SOD1 mice leads to limited SaCas9 expression in GFAP+ astrocytes in the spinal cord.
  • fig. S8. CRISPR-Cas9–mediated genome editing reduced mutant SOD1 protein in G93A-SOD1 mice.
  • fig. S9. Genome editing did not affect mouse SOD1 protein in G93A-SOD1 mice.
  • fig. S10. Background modification at candidate OT sites in CRISPR-treated G93A-SOD1 mice.
  • fig. S11. G93A-SOD1 mice treated with AAV9-SaCas9-hSOD1 lose weight at a slower rate after disease onset compared to control mice.
  • fig. S12. Systemic administration of AAV9-SaCas9-hSOD1 to neonatal G93A-SOD1 mice did not delay the rate of disease progression.
  • fig. S13. G93A-SOD1 mice injected with AAV9-SaCas9-SaCas9 had limited SaCas9 expression in GFAP+ astrocytes at end stage.
  • fig. S14. Mutant SOD1 inclusion bodies were visible in end-stage spinal cord sections from CRISPR-treated G93A-SOD1 mice.
  • table S1. Oligonucleotides used in this study.
  • table S2. External primers for MiSeq analysis.
  • table S3. Internal primers for MiSeq analysis.

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