Supplementary Materials

The PDF file includes:

  • Materials and Methods
  • Fig. S1. Phylogenetic analysis of AAV Rep proteins isolated from human liver (AAV-hu.Lvr01, AAV-hu.Lvr 02, AAV-hu.Lvr 05, and AAV-hu.Lvr 07).
  • Fig. S2. Murine liver transduction comparison of AAV-hu.Lvr capsids in the xenograft liver model (hFRG #171).
  • Fig. S3. Multiple sequence alignment of prototypical AAV2 and AAV13 with related hu.Lvr capsids.
  • Fig. S4. HiTrap Heparin column binding assay of prototypical AAV2.
  • Fig. S5. HiTrap Heparin column binding assay of AAV-hu.Lvr01.
  • Fig. S6. HiTrap Heparin column binding assay of AAV-hu.Lvr02.
  • Fig. S7. HiTrap Heparin column binding assay of AAV-hu.Lvr03.
  • Fig. S8. HiTrap Heparin column binding assay of AAV-hu.Lvr04.
  • Fig. S9. HiTrap Heparin column binding assay of AAV-hu.Lvr05.
  • Fig. S10. HiTrap Heparin column binding assay of AAV-hu.Lvr06.
  • Fig. S11. HiTrap Heparin column binding assay of AAV-hu.Lvr07.
  • Fig. S12. Representative controls for the in vitro adaptation.
  • Fig. S13. Population shift of hu.Lvr02 N469 and E555 during in vitro adaptation.
  • Fig. S14. HiTrap Heparin column binding assay of caAAV-hu.Lvr07E530K.
  • Fig. S15. HiTrap Heparin column binding assay of caAAV-hu.Lvr07G593R.
  • Fig. S16. HiTrap Heparin column binding assay of caAAV-hu.Lvr02D469N+E555K.
  • Fig. S17. In vitro heparin competition assay of hu.Lvr02 and culture-adapted AAV-hu.Lvr02D469N+E555K.
  • Fig. S18. HiTrap Heparin column binding assay of AAV13.
  • Fig. S19. HiTrap Heparin column binding assay of AAV3B.
  • Fig. S20. HiTrap Heparin column binding assay of AAV2-R588I.
  • Fig. S21. HiTrap Heparin column binding assay of AAV2-R588T.
  • Fig. S22. HiTrap Heparin column binding assay of AAV2-R487Q.
  • Fig. S23. Murine liver transduction comparison of AAV2 capsid variants in the xenograft liver model (hFRG #201).
  • Fig. S24. AAV yield comparison of RC-AAV2 and RC-AAV2-R588I.
  • Fig. S25. Immunohistochemical staining of engrafted human hepatocytes with human glyceraldehyde-3-phosphate dehydrogenase and human fumarlyacetoacetase hydrolase (FAH).
  • Fig. S26. ddPCR analysis of chimeric liver lysate of hFRG #445.
  • Fig. S27. Murine liver transduction comparison of AAV-hu.Lvr07 and its adapted (E530K) variants in the xenograft liver model (hFRG #164).
  • Table S1. Primer sequences.
  • Table S2. hFRG individual mouse data.
  • Table S3. In vivo comparison of hu.Lvr variants—NGS Read Summary.
  • Table S4. In vivo comparison of culture-adapted hu.Lvr07 variant—NGS Read Summary.
  • Table S5. In vivo comparison of AAV2 variants—NGS Read Summary.
  • Table S6. Summary of HiTrap Heparin column binding assay.
  • Table S7. Clonal analysis of AAV2 in vivo adapted variants round 1.
  • Table S8. Clonal analysis of in vivo adapted variants round 2.
  • Table S9. Clonal analysis of in vivo adapted variants round 3.

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Other Supplementary Material for this manuscript includes the following:

  • Data file S1 (Microsoft Excel format). Statistical analyses.
  • Data file S2 (Microsoft Excel format). NGS data in tabular format for Figures 1 (B and C), 2 (G and H), and 4 (A and B).