Science Advances

Supplementary Materials

This PDF file includes:

  • Fig. S1. Alkaline denaturing gel electrophoresis confirms integrity of AAV vectors.
  • Fig. S2. Systemic delivery of CRISPR-Cas9 genome editing components by single-stranded AAV vector to ∆Ex44 mice rescues dystrophin expression.
  • Fig. S3. Whole muscle scanning of immunohistochemistry of TA, triceps, diaphragm, and heart of CRISPR-Cas9–corrected ∆Ex44 mice.
  • Fig. S4. Western blot analysis of skeletal muscles and heart of ∆Ex44 mice treated with ssAAV-packaged CRISPR-Cas9 genome editing components.
  • Fig. S5. Muscle histology of ΔEx44 mice after systemic delivery of AAV expressing CRISPR-Cas9 genome editing components.
  • Fig. S6. Whole-muscle scanning of H&E staining of TA, triceps, diaphragm, and heart of CRISPR-Cas9–corrected ∆Ex44 mice.
  • Fig. S7. Serum CK analysis of CRISPR-Cas9–corrected ∆Ex44 mice.
  • Fig. S8. ∆Ex44 mice express more Cas9 and sgRNA transcripts after systemic delivery of scAAV-packaged sgRNA.
  • Table S1. Deep sequencing analysis of CRISPR-Cas9–corrected ∆Ex44 mice.
  • Table S2. Primers used in this study.

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