Science Advances

Supplementary Materials

This PDF file includes:

  • Fig. S1. Analysis of sgRNAs that target the splice acceptor or donor sites for exons 43 and 45.
  • Fig. S2. Characterization of the ΔEx44 mouse line.
  • Fig. S3. Intramuscular AAV9 delivery of gene editing components rescues dystrophin expression.
  • Fig. S4. Analysis of top 10 potential off-target sites.
  • Fig. S5. Correction of ΔEx44 mice by systemic delivery of AAV9 expressing gene editing components.
  • Fig. S6. Western blot analysis of corrected ΔEx44 mice by systemic delivery of AAV9 expressing gene editing components.
  • Fig. S7. Histology of ΔEx44 mice after systemic delivery of AAV9 expressing gene editing components.
  • Fig. S8. Quantification of histological improvement and qPCR analysis of corrected ΔEx44 DMD mice.
  • Fig. S9. Histological analysis showing dystrophin restoration in the EDL muscle of corrected ΔEx44 DMD mice.
  • Table S1. Primer sequences and media components.

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