Characterization and quantification of adeno-associated virus capsid-loading states by multi-wavelength analytical ultracentrifugation with UltraScan

Nanomedicine (Lond). 2023 Sep;18(22):1519-1534. doi: 10.2217/nnm-2023-0156. Epub 2023 Oct 25.

Abstract

Aim: We present multi-wavelength (MW) analytical ultracentrifugation (AUC) methods offering superior accuracy for adeno-associated virus characterization and quantification. Methods: Experimental design guidelines are presented for MW sedimentation velocity and analytical buoyant density equilibrium AUC. Results: Our results were compared with dual-wavelength AUC, transmission electron microscopy and mass photometry. In contrast to dual-wavelength AUC, MW-AUC correctly quantifies adeno-associated virus capsid ratios and identifies contaminants. In contrast to transmission electron microscopy, partially filled capsids can also be detected and quantified. In contrast to mass photometry, first-principle results are obtained. Conclusion: Our study demonstrates the improved information provided by MW-AUC, highlighting the utility of several recently integrated UltraScan programs, and reinforces AUC as the gold-standard analysis for viral vectors.

Keywords: UltraScan; adeno-associated virus; analytical buoyant density gradient equilibrium; gene therapy; multi-wavelength analytical ultracentrifugation; sedimentation velocity; viral capsid quantification; viral vector.

Publication types

  • Research Support, U.S. Gov't, Non-P.H.S.
  • Research Support, Non-U.S. Gov't
  • Research Support, N.I.H., Extramural

MeSH terms

  • Capsid*
  • Dependovirus* / genetics
  • Genetic Vectors
  • Microscopy, Electron, Transmission
  • Ultracentrifugation / methods