Distinct classes of proteasome-modulating agents cooperatively augment recombinant adeno-associated virus type 2 and type 5-mediated transduction from the apical surfaces of human airway epithelia

J Virol. 2004 Mar;78(6):2863-74. doi: 10.1128/jvi.78.6.2863-2874.2004.

Abstract

Tripeptidyl aldehyde proteasome inhibitors have been shown to effectively increase viral capsid ubiquitination and transduction of recombinant adeno-associated virus type 2 (rAAV-2) and rAAV-5 serotypes. In the present study we have characterized a second class of proteasome-modulating agents (anthracycline derivatives) for their ability to induce rAAV transduction. The anthracycline derivatives doxorubicin and aclarubicin were chosen for analysis because they have been shown to interact with the proteasome through a mechanism distinct from that of tripeptidyl aldehydes. Our studies demonstrated that doxorubicin and aclarubicin also significantly augmented rAAV transduction in airway cell lines, polarized human airway epithelia, and mouse lungs. Both tripeptidyl aldehyde and anthracycline proteasome-modulating agents similarly augmented nuclear accumulation of rAAV in A549 and IB3 airway cell lines. However, these two cell types demonstrated cell specificity in the ability of N-acetyl-L-leucyl-L-leucyl-L-norleucine (LLnL) or doxorubicin to augment rAAV transduction. Interestingly, the combined administration of LLnL and doxorubicin resulted in substantially increased transduction (>2,000-fold) following apical infection of human polarized epithelia with either rAAV-2 or rAAV-5. In summary, the cell type specificity of LLnL and doxorubicin to induce rAAV transduction, together with the ability of these compounds to synergistically enhance rAAV transduction in polarized airway epithelial induction, suggests that these two classes of compounds likely modulate different proteasome functions that affect rAAV transduction. Findings from this study provide new insights into how modulation of proteasome function can be effectively used to augment rAAV transduction in airway epithelia for gene therapy of cystic fibrosis.

Publication types

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

MeSH terms

  • Aclarubicin / pharmacology
  • Animals
  • Cell Line
  • Cell Polarity
  • Cysteine Endopeptidases
  • Cysteine Proteinase Inhibitors / pharmacology
  • Cystic Fibrosis
  • Dependovirus / drug effects*
  • Dependovirus / genetics
  • Dependovirus / pathogenicity
  • Doxorubicin / pharmacology
  • Epithelial Cells / virology*
  • Genetic Therapy
  • Humans
  • Leupeptins / pharmacology
  • Lung / cytology
  • Lung / virology
  • Mice
  • Mice, Inbred C57BL
  • Multienzyme Complexes / antagonists & inhibitors*
  • Parvoviridae Infections / virology
  • Proteasome Endopeptidase Complex
  • Recombination, Genetic
  • Transduction, Genetic*

Substances

  • Cysteine Proteinase Inhibitors
  • Leupeptins
  • Multienzyme Complexes
  • Aclarubicin
  • Doxorubicin
  • Cysteine Endopeptidases
  • Proteasome Endopeptidase Complex
  • benzyloxycarbonylleucyl-leucyl-leucine aldehyde