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Liver induced transgene tolerance with AAV vectors.

Authors
  • Keeler, Geoffrey D1
  • Markusic, David M1
  • Hoffman, Brad E2
  • 1 Department of Pediatrics, Div. Cell and Molecular Therapy, University of Florida, United States. , (United States)
  • 2 Department of Pediatrics, Div. Cell and Molecular Therapy, University of Florida, United States; Department of Neuroscience, University of Florida, United States. Electronic address: [email protected] , (United States)
Type
Published Article
Journal
Cellular Immunology
Publisher
Elsevier
Publication Date
Aug 01, 2019
Volume
342
Pages
103728–103728
Identifiers
DOI: 10.1016/j.cellimm.2017.12.002
PMID: 29576315
Source
Medline
Keywords
Language
English
License
Unknown

Abstract

Immune tolerance is a vital component of immunity, as persistent activation of immune cells causes significant tissue damage and loss of tolerance leads to autoimmunity. Likewise, unwanted immune responses can occur in inherited disorders, such as hemophilia and Pompe disease, in which patients lack any expression of protein, during treatment with enzyme replacement therapy, or gene therapy. While the liver has long been known as being tolerogenic, it was only recently appreciated in the last decade that liver directed adeno-associated virus (AAV) gene therapy can induce systemic tolerance to a transgene. In this review, we look at the mechanisms behind liver induced tolerance, discuss different factors influencing successful tolerance induction with AAV, and applications where AAV mediated tolerance may be helpful. Copyright © 2017 Elsevier Inc. All rights reserved.

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