A TLR and non-TLR mediated innate response to lentiviruses restricts hepatocyte entry and can be ameliorated by pharmacological blockade.

TitleA TLR and non-TLR mediated innate response to lentiviruses restricts hepatocyte entry and can be ameliorated by pharmacological blockade.
Publication TypeJournal Article
Year of Publication2012
AuthorsAgudo, J, Ruzo, A, Kitur, K, Sachidanandam, R, J Blander, M, Brown, BD
JournalMol Ther
Volume20
Issue12
Pagination2257-67
Date Published2012 Dec
ISSN1525-0024
KeywordsAdaptor Proteins, Vesicular Transport, Animals, Cells, Cultured, Flow Cytometry, Hepatocytes, Immunity, Innate, Immunohistochemistry, Lentivirus, Liver, Mice, Mice, Knockout, Myeloid Differentiation Factor 88, Toll-Like Receptors
Abstract

Lentiviral vector (LV)-mediated gene transfer is a promising method of gene therapy. We previously reported that systemic injection of HIV-based LV triggers a transient inflammatory response. Here, we carried out studies to better characterize this response, and to develop a strategy to overcome the adverse effects of interferon (IFN) on LV-mediated gene transfer. We profiled gene expression in the liver after LV administration using deep-sequencing (RNA-seq), and identified several innate response pathways. We examined the response to LV in MyD88-TRIF knockout mice, which are incapable of toll-like receptor (TLR) signaling. Unexpectedly, the IFN response to LV was not reduced in the liver indicating that a non-TLR pathway can recognize LV in this organ. Indeed, blocking reverse transcription with azidothymidine (AZT) reduced the IFN response only in the liver, suggesting that proviral DNA can be a trigger. To block the inflammatory response, we pretreated mice with a short course of dexamethasone (Dex). At 4 hours post-treatment, all the IFN-induced genes were normalized. By blocking the inflammatory response, hepatocyte transduction was dramatically increased, which in turn doubled the level of human factor IX (FIX) produced by a hepatocyte-specific LV. Our studies uncover new insights into LV-induced immune responses in the liver, and provide a means to increase the safety and efficiency of LV-mediated gene transfer.

DOI10.1038/mt.2012.150
Alternate JournalMol. Ther.
PubMed ID22871668
PubMed Central IDPMC3519984
Grant ListDP2 DK083052 / DK / NIDDK NIH HHS / United States
R01 AI073899 / AI / NIAID NIH HHS / United States
1R01AI073899-01 / AI / NIAID NIH HHS / United States
DP2DK083052-01 / DK / NIDDK NIH HHS / United States