Nicholas D Weber, Daniel Stone, Keith R Jerome Molecular Therapy

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TALENs Targeting HBV: Designer Endonuclease Therapies for Viral Infections Nicholas D Weber, Daniel Stone, Keith R Jerome Molecular Therapy Volume 21, Issue 10, Pages 1819-1821 (October 2013) DOI: 10.1038/mt.2013.208 Copyright © 2013 The American Society of Gene & Cell Therapy Terms and Conditions

Figure 1 Inhibition of HBV replication. Currently approved antivirals include reverse transcriptase inhibitors (RTi) and the immune system modulator interferon-α2a. Covalently closed circular DNA (cccDNA) is not affected by these antiviral drugs and remains in the nucleus throughout the lifetime of an infected cell. Targeted endonucleases promote disruption of cccDNA, which prevents viral replication by blocking expression of functional viral proteins. CRISPR, clustered regularly interspaced short palindromic repeats; ER, endoplasmic reticulum; HBcAg, hepatitis B core antigen; HBsAg, hepatitis B surface antigen; HE, homing endonuclease; pgRNA, pregenomic RNA; rcDNA, relaxed circular DNA; TALEN, transcription activator–like effector nuclease; ZFN, zinc-finger nuclease. Molecular Therapy 2013 21, 1819-1821DOI: (10.1038/mt.2013.208) Copyright © 2013 The American Society of Gene & Cell Therapy Terms and Conditions