Capsid-specific removal of circulating antibodies to adeno-associated virus vectors

Sci Rep. 2020 Jan 21;10(1):864. doi: 10.1038/s41598-020-57893-z.

Abstract

Neutralizing antibodies directed against adeno-associated virus (AAV) are commonly found in humans. In seropositive subjects, vector administration is not feasible as antibodies neutralize AAV vectors even at low titers. Consequently, a relatively large proportion of humans is excluded from enrollment in clinical trials and, similarly, vector redosing is not feasible because of development of high-titer antibodies following AAV vector administration. Plasmapheresis has been proposed as strategy to remove anti-AAV antibodies from the bloodstream. Although safe and relatively effective, the technology has some limitations mainly related to the nonspecific removal of all circulating IgG. Here we developed an AAV-specific plasmapheresis column which was shown to efficiently and selectively deplete anti-AAV antibodies without depleting the total immunoglobulin pool from plasma. We showed the nearly complete removal of anti-AAV antibodies from high titer purified human IgG pools and plasma samples, decreasing titers to levels that allow AAV vector administration in mice. These results provide proof-of-concept of a method for the AAV-specific depletion of neutralizing antibodies in the setting of in vivo gene transfer.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Animals
  • Antibodies, Neutralizing / isolation & purification*
  • Antibodies, Viral / isolation & purification*
  • Capsid*
  • Dependovirus / immunology*
  • Gene Transfer Techniques
  • Genetic Vectors / immunology*
  • Humans
  • Immunoglobulin G / isolation & purification*
  • Mice
  • Plasmapheresis / methods*

Substances

  • Antibodies, Neutralizing
  • Antibodies, Viral
  • Immunoglobulin G