Supplementary Materials

The PDF file includes:

  • Fig. S1. Design and mechanism of action of rAAV5-miHTT leading to HTT lowering in the cell.
  • Fig. S2. Brain dissection scheme for bioanalytics.
  • Fig. S3. VG copies in deep brain regions of tgHD minipigs at 6 and 12 months after rAAV5-miHTT administration in the caudate and putamen.
  • Fig. S4. Correlation between miHTT expression and mHTT protein lowering (as percentage from control), 12 months after rAAV5-miHTT administration.
  • Fig. S5. HTT protein concentrations in the CSF of tgHD minipigs increase with age, as determined by two independent immunoassays.
  • Fig. S6. No differences in CSF NFL between wild-type and tgHD minipigs up to 4 years of age.
  • Table S1. Statistical analysis of mHTT in the CSF of tgHD minipigs (control versus treated).
  • Table S2. Experimental approach for intrastriatal miRNA-based gene therapy in tgHD minipigs.
  • Legend for data file S1

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Other Supplementary Material for this manuscript includes the following:

  • Data file S1. Raw data (provided as supplementary Excel file).