Disruption of Microtubules Post-Virus Entry Enhances Adeno-Associated Virus Vector Transduction

Hum Gene Ther. 2016 Apr;27(4):309-24. doi: 10.1089/hum.2016.008.

Abstract

Perinuclear retention of viral particles is a poorly understood phenomenon observed during many virus infections. In this study, we investigated whether perinuclear accumulation acts as a barrier to limit recombinant adeno-associated virus (rAAV) transduction. After nocodazole treatment to disrupt microtubules at microtubule-organization center (MT-MTOC) after virus entry, we observed higher rAAV transduction. To elucidate the role of MT-MTOC in rAAV infection and study its underlying mechanisms, we demonstrated that rAAV's perinuclear localization was retained by MT-MTOC with fluorescent analysis, and enhanced rAAV transduction from MT-MTOC disruption was dependent on the rAAV capsid's nuclear import signals. Interestingly, after knocking down RhoA or inhibiting its downstream effectors (ROCK and Actin), MT-MTOC disruption failed to increase rAAV transduction or nuclear entry. These data suggest that enhancement of rAAV transduction is the result of increased trafficking to the nucleus via the RhoA-ROCK-Actin pathway. Ten-fold higher rAAV transduction was also observed by disrupting MT-MTOC in brain, liver, and tumor in vivo. In summary, this study indicates that virus perinuclear accumulation at MT-MTOC is a barrier-limiting parameter for effective rAAV transduction and defines a novel defense mechanism by which host cells restrain viral invasion.

Publication types

  • Research Support, N.I.H., Extramural

MeSH terms

  • Animals
  • Cell Cycle Checkpoints / drug effects
  • Cell Nucleus / drug effects
  • Cell Nucleus / virology
  • Dependovirus / metabolism*
  • Genetic Vectors / metabolism*
  • HEK293 Cells
  • HeLa Cells
  • Humans
  • Mice, Inbred BALB C
  • Mice, SCID
  • Microtubule-Organizing Center / drug effects
  • Microtubule-Organizing Center / metabolism
  • Microtubules / drug effects
  • Microtubules / metabolism*
  • Nocodazole / pharmacology
  • Promoter Regions, Genetic / genetics
  • Signal Transduction / drug effects
  • Transduction, Genetic*
  • Virus Internalization* / drug effects

Substances

  • Nocodazole