Biodistribution and retargeting of FX-binding ablated adenovirus serotype 5 vectors

Blood. 2010 Oct 14;116(15):2656-64. doi: 10.1182/blood-2009-12-260026. Epub 2010 Jul 7.

Abstract

A major limitation for adenoviral transduction in vivo is the profound liver tropism of adenovirus type 5 (Ad5). Recently, we demonstrated that coagulation factor X (FX) binds to Ad5-hexon protein at high affinity to mediate hepatocyte transduction after intravascular delivery. We developed novel genetically FX-binding ablated Ad5 vectors with lower liver transduction. Here, we demonstrate that FX-binding ablated Ad5 predominantly localize to the liver and spleen 1 hour after injection; however, they had highly reduced liver transduction in both control and macrophage-depleted mice compared with Ad5. At high doses in macrophage-depleted mice, FX-binding ablated vectors transduced the spleen more efficiently than Ad5. Immunohistochemical studies demonstrated transgene colocalization with CD11c(+), ER-TR7(+), and MAdCAM-1(+) cells in the splenic marginal zone. Systemic inflammatory profiles were broadly similar between FX-binding ablated Ad5 and Ad5 at low and intermediate doses, although higher levels of several inflammatory proteins were observed at the highest dose of FX-binding ablated Ad5. Subsequently, we generated a FX-binding ablated virus containing a high affinity Ad35 fiber that mediated a significant improvement in lung/liver ratio in macrophage-depleted CD46(+) mice compared with controls. Therefore, this study documents the biodistribution and reports the retargeting capacity of FX binding-ablated Ad5 vectors in vitro and in vivo.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Adenoviruses, Human / classification
  • Adenoviruses, Human / genetics*
  • Adenoviruses, Human / metabolism*
  • Animals
  • Capsid Proteins / genetics
  • Capsid Proteins / metabolism*
  • Chemokines / metabolism
  • Cytokines / metabolism
  • Factor X / metabolism*
  • Genetic Vectors*
  • Humans
  • Inflammation Mediators / metabolism
  • Liver / metabolism
  • Liver / virology
  • Lung / metabolism
  • Lung / virology
  • Male
  • Mice
  • Mice, Transgenic
  • Protein Binding
  • Recombinant Proteins / genetics
  • Recombinant Proteins / metabolism
  • Serotyping
  • Spleen / metabolism
  • Spleen / virology
  • Time Factors
  • Tissue Distribution
  • Transduction, Genetic
  • beta-Galactosidase / genetics
  • beta-Galactosidase / metabolism

Substances

  • Capsid Proteins
  • Chemokines
  • Cytokines
  • Inflammation Mediators
  • Recombinant Proteins
  • hexon capsid protein, Adenovirus
  • Factor X
  • beta-Galactosidase