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Recent Advances in Use of Gene Therapy to Treat Hepatitis B Virus Infection

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Gene Therapy for HIV and Chronic Infections

Part of the book series: Advances in Experimental Medicine and Biology ((ASGCT,volume 848))

Abstract

Chronic infection with hepatitis B virus (HBV) occurs in approximately 5 % of the world’s human population and persistence of the virus is associated with serious complications of cirrhosis and liver cancer. Currently available treatments are modestly effective and advancing novel therapeutic strategies is a medical priority. Stability of the viral cccDNA replication intermediate is a major factor that has impeded the development of therapies that are capable of eliminating chronic infection. Recent advances that employ gene therapy strategies offer useful advantages over current therapeutics. Silencing of HBV gene expression by harnessing the RNA interference pathway has been shown to be highly effective in cell culture and in vivo. However, a potential limitation of this approach is that the post-transcriptional mechanism of gene silencing does not disable cccDNA. Early results using designer transcription activator-like effector nucleases (TALENs) and repressor TALEs (rTALEs) have shown potential as a mode of inactivating cccDNA. In this article, we review the recent advances that have been made in HBV gene therapy, with a particular emphasis on the potential anti-HBV therapeutic utility of designed sequence-specific DNA binding proteins and their derivatives.

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Acknowledgements

Work in the authors’ laboratory is generously supported by funding from the National Research Foundation (NRF, GUNs 81768, 81692, 68339, 85981 & 77954) of South Africa, South African Medical Research Council (MRC), Poliomyelitis Research Foundation (PRF) and from the German Research Foundation (DFG).

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Authors and Affiliations

  1. Institute for Cell and Gene Therapy and Center for Chronic Immunodeficiency, University Medical Center Freiburg, Freiburg, Germany

    Kristie Bloom Ph.D.

  2. Antiviral Gene Therapy Research Unit, School of Pathology, Health Sciences Faculty, University of the Witwatersrand, Private Bag 3, WITS 2050, Johannesburg, South Africa

    Abdullah Ely Ph.D. & Patrick Arbuthnot M.D., Ph.D.

Authors
  1. Kristie Bloom Ph.D.
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  2. Abdullah Ely Ph.D.
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  3. Patrick Arbuthnot M.D., Ph.D.
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Correspondence to Patrick Arbuthnot M.D., Ph.D. .

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Editors and Affiliations

  1. University of Amsterdam, Amsterdam, The Netherlands

    Ben Berkhout

  2. The Wistar Institute, University of Pennsylvania, Philadelphia, Pennsylvania, USA

    Hildegund C.J. Ertl

  3. University of the Witwatersrand, Johannesburg, South Africa

    Marc S. Weinberg

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© 2015 American Society of Gene and Cell Therapy

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Bloom, K., Ely, A., Arbuthnot, P. (2015). Recent Advances in Use of Gene Therapy to Treat Hepatitis B Virus Infection. In: Berkhout, B., Ertl, H., Weinberg, M. (eds) Gene Therapy for HIV and Chronic Infections. Advances in Experimental Medicine and Biology(), vol 848. Springer, New York, NY. https://doi.org/10.1007/978-1-4939-2432-5_2

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