Supplementary Materials

Supplementary Material for:

An miRNA-mediated therapy for SCA6 blocks IRES-driven translation of the CACNA1A second cistron

Yu Miyazaki, Xiaofei Du, Shin-ichi Muramatsu, Christopher M. Gomez*

*Corresponding author. E-mail: cgomez{at}neurology.bsd.uchicago.edu

Published 13 July 2016, Sci. Transl. Med. 8, 347ra94 (2016)
DOI: 10.1126/scitranslmed.aaf5660

This PDF file includes:

  • Fig. S1. Schematic representation of AAV9 vectors and AAV9 transduction efficiency into Purkinje cells.
  • Fig. S2. Histopathological examination of the AAV9-injected mouse cerebral cortex and hippocampus.
  • Fig. S3. The long-term follow-up of AAV9-injected mouse behavioral phenotypes and CACNA1A IRES–driven α1ACT expression in mice.
  • Fig. S4. Stem-loop structure of CACNA1A IRES and the predicted binding sites of miR-711, miR-3191-5p, and miR-4786-3p.
  • Fig. S5. Effects of miR-3191-5p on mutated CACNA1A IRES templates.
  • Fig. S6. Effects of eIF4AII and eIF4GII on mutated CACNA1A IRES templates in the presence or absence of miR-3191-5p.
  • Fig. S7. Cotransduction of AAV9-α1ACT-Q33 with either AAV9–miR-mock or AAV9–miR-3191-5p.
  • Fig. S8. The long-term follow-up of therapeutic effect of miR-3191-5p on mouse behavioral phenotypes and CACNA1A IRES–driven α1ACTSCA6 expression in mice.
  • Fig. S9. Potential mouse mRNAs targeted by human miR-3191-5p.
  • Fig. S10. Histopathological examination of the brain, cerebellum, heart, lung, liver, and kidney of wild-type mice injected with AAV–miR-3191-5p.
  • Legend for table S1
  • Legends for movies S1 to S5

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Other Supplementary Material for this manuscript includes the following:

  • Table S1 (Microsoft Excel format). Source data.
  • Movie S1 (.mov format). DigiGait video of an 8-week-old AAV9-GFP mouse on treadmill.
  • Movie S2 (.mov format). DigiGait video of an 8-week-old AAV9-α1ACT-Q11 mouse on treadmill.
  • Movie S3 (.mov format). DigiGait video of an 8-week-old AAV9-α1ACT-Q33 mouse on treadmill.
  • Movie S4 (.mov format). DigiGait video of an 8-week-old AAV9–Q33–miRmock mouse on treadmill.
  • Movie S5 (.mov format). DigiGait video of an 8-week-old AAV9–Q33–miR- 3191-5p mouse on treadmill.