Supplementary Materials

Supplementary Material for:

Systemic administration of the antisense oligonucleotide NS-065/NCNP-01 for skipping of exon 53 in patients with Duchenne muscular dystrophy

Hirofumi Komaki, Tetsuya Nagata, Takashi Saito, Satoru Masuda, Eri Takeshita, Masayuki Sasaki, Hisateru Tachimori, Harumasa Nakamura, Yoshitsugu Aoki, Shin'ichi Takeda*

*Corresponding author. Email: takeda{at}ncnp.go.jp

Published 18 April 2018, Sci. Transl. Med. 10, eaan0713 (2018)
DOI: 10.1126/scitranslmed.aan07131

This PDF file includes:

  • Appendix S1. Clinical study protocol synopsis
  • Materials and Methods
  • Fig. S1. Results of the in vitro dystrophin assay.
  • Fig. S2. Preliminary validation of immunofluorescence analysis.
  • Fig. S3. Preliminary validation of Western blotting analysis.
  • Table S1. Change in blood creatine kinase over time.
  • Table S2. Urinary protein in 24-hour pooled urine samples.

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