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

  • Kristie Bloom
  • Abdullah Ely
  • Patrick Arbuthnot
Part of the Advances in Experimental Medicine and Biology book series (AEMB, volume 848)


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.


HBV cccDNA RNAi Designer nucleases TALENs Zinc finger nucleases Repressor TALEs Targeted disruption Site-directed mutagenesis 



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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Copyright information

© American Society of Gene and Cell Therapy 2015

Authors and Affiliations

  • Kristie Bloom
    • 1
  • Abdullah Ely
    • 2
  • Patrick Arbuthnot
    • 2
  1. 1.Institute for Cell and Gene Therapy and Center for Chronic ImmunodeficiencyUniversity Medical Center FreiburgFreiburgGermany
  2. 2.Antiviral Gene Therapy Research Unit, School of Pathology, Health Sciences FacultyUniversity of the WitwatersrandJohannesburgSouth Africa

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