Inhibition of replication of hepatitis B virus using transcriptional repressors that target the viral DNA.


Journal

BMC infectious diseases
ISSN: 1471-2334
Titre abrégé: BMC Infect Dis
Pays: England
ID NLM: 100968551

Informations de publication

Date de publication:
12 Sep 2019
Historique:
received: 08 06 2019
accepted: 03 09 2019
entrez: 13 9 2019
pubmed: 13 9 2019
medline: 15 10 2019
Statut: epublish

Résumé

Chronic infection with hepatitis B virus (HBV) is a serious global health problem. Persistence of the virus occurs as a result of stability of the replication intermediate comprising covalently closed circular DNA (cccDNA). Development of drugs that are capable of disabling this cccDNA is vital. To investigate an epigenetic approach to inactivating viral DNA, we engineered transcriptional repressors that comprise an HBV DNA-binding domain of transcription activator like effectors (TALEs) and a fused Krüppel Associated Box (KRAB). These repressor TALEs (rTALEs) targeted the viral surface open reading frame and were placed under transcription control of constitutively active or liver-specific promoters. Evaluation in cultured cells and following hydrodynamic injection of mice revealed that the rTALEs significantly inhibited production of markers of HBV replication without evidence of hepatotoxicity. Increased methylation of HBV DNA at CpG island II showed that the rTALEs caused intended epigenetic modification. Epigenetic modification of HBV DNA is a new and effective means of inactivating the virus in vivo. The approach has therapeutic potential and avoids potentially problematic unintended mutagenesis of gene editing.

Sections du résumé

BACKGROUND BACKGROUND
Chronic infection with hepatitis B virus (HBV) is a serious global health problem. Persistence of the virus occurs as a result of stability of the replication intermediate comprising covalently closed circular DNA (cccDNA). Development of drugs that are capable of disabling this cccDNA is vital.
METHODS METHODS
To investigate an epigenetic approach to inactivating viral DNA, we engineered transcriptional repressors that comprise an HBV DNA-binding domain of transcription activator like effectors (TALEs) and a fused Krüppel Associated Box (KRAB). These repressor TALEs (rTALEs) targeted the viral surface open reading frame and were placed under transcription control of constitutively active or liver-specific promoters.
RESULTS RESULTS
Evaluation in cultured cells and following hydrodynamic injection of mice revealed that the rTALEs significantly inhibited production of markers of HBV replication without evidence of hepatotoxicity. Increased methylation of HBV DNA at CpG island II showed that the rTALEs caused intended epigenetic modification.
CONCLUSIONS CONCLUSIONS
Epigenetic modification of HBV DNA is a new and effective means of inactivating the virus in vivo. The approach has therapeutic potential and avoids potentially problematic unintended mutagenesis of gene editing.

Identifiants

pubmed: 31510934
doi: 10.1186/s12879-019-4436-y
pii: 10.1186/s12879-019-4436-y
pmc: PMC6739920
doi:

Substances chimiques

DNA, Circular 0
DNA, Viral 0
Repressor Proteins 0

Types de publication

Journal Article

Langues

eng

Sous-ensembles de citation

IM

Pagination

802

Subventions

Organisme : South African National Research Foundation
ID : 81768, 81692, 68339, 85981 & 77954
Organisme : South African Poliomyelitis Research Foundation
ID : Not applicable
Organisme : German Federal Ministry for Education and Research
ID : HBVTALE-10DG15005, BMBF-01EO0803

Références

Hepatol Int. 2016 Jan;10(1):1-98
pubmed: 26563120
Mol Ther. 2007 Mar;15(3):534-41
pubmed: 17213835
Hepatology. 2016 Jan;63(1):261-83
pubmed: 26566064
J Virol. 1995 Oct;69(10):6158-69
pubmed: 7666518
J Infect Dis. 2010 Sep 1;202(5):700-4
pubmed: 20653444
PLoS One. 2014 Oct 22;9(10):e110442
pubmed: 25337821
J Virol. 1992 Jul;66(7):4107-16
pubmed: 1602535
J Med Virol. 2009 Jul;81(7):1177-83
pubmed: 19475606
Curr Protoc Mol Biol. 2004 Sep;Chapter 4:Unit 4.9
pubmed: 18265351
Med Chem. 2015;11(8):710-6
pubmed: 25985859
Epigenetics. 2011 Jun;6(6):720-6
pubmed: 21546797
Microbiol Immunol. 2016 Jan;60(1):17-25
pubmed: 26616333
Nucleic Acids Res. 1991 Aug 11;19(15):4139-45
pubmed: 1870969
J Hepatol. 2016 Sep;65(3):483-9
pubmed: 27132172
Biochem Biophys Res Commun. 2000 Feb 24;268(3):728-33
pubmed: 10679273
Science. 2018 Nov 30;362(6418):984-987
pubmed: 30498108
Mol Ther. 2013 Oct;21(10):1889-97
pubmed: 23883864
Nat Methods. 2015 Dec;12(12):1143-9
pubmed: 26501517
J Viral Hepat. 2008 Feb;15(2):103-7
pubmed: 18184192
Bioorg Med Chem. 2013 Oct 15;21(20):6145-55
pubmed: 23743442
J Control Release. 2015 Jul 10;209:198-206
pubmed: 25937322
Nat Methods. 2015 May;12(5):401-403
pubmed: 25775043
FEBS J. 2017 Nov;284(21):3550-3572
pubmed: 28457020
PLoS One. 2013;8(2):e56711
pubmed: 23451072
Genes (Basel). 2018 Apr 12;9(4):
pubmed: 29649127
Nature. 2018 Dec;564(7734):24-26
pubmed: 30518904
Hepatology. 2017 Aug;66(2):398-415
pubmed: 28236308
PLoS One. 2016 Sep 22;11(9):e0163555
pubmed: 27658112
Lancet. 2016 Sep 10;388(10049):1081-1088
pubmed: 27394647
Proc Natl Acad Sci U S A. 2009 Nov 24;106(47):19975-9
pubmed: 19906987
Proc Natl Acad Sci U S A. 2002 Oct 15;99(21):13825-30
pubmed: 12374864
Genome Biol. 2003;4(10):231
pubmed: 14519192
Oncol Rep. 2017 May;37(5):2811-2818
pubmed: 28339081
J Hepatol. 2017 Aug;67(2):370-398
pubmed: 28427875
Nat Methods. 2012 Jul;9(7):671-5
pubmed: 22930834
Mol Ther. 2016 Apr;24(4):671-7
pubmed: 26916283

Auteurs

Kristie Bloom (K)

Wits/SAMRC Antiviral Gene Therapy Research Unit, School of Pathology, Faculty of Health Science, University of the Witwatersrand, Private Bag 3, WITS, Johannesburg, 2050, South Africa.

Haajira Kaldine (H)

Wits/SAMRC Antiviral Gene Therapy Research Unit, School of Pathology, Faculty of Health Science, University of the Witwatersrand, Private Bag 3, WITS, Johannesburg, 2050, South Africa.

Toni Cathomen (T)

Institute for Transfusion Medicine and Gene Therapy, Medical Center - University of Freiburg, Freiburg, Germany.
Faculty of Medicine, University of Freiburg, Freiburg, Germany.

Claudio Mussolino (C)

Institute for Transfusion Medicine and Gene Therapy, Medical Center - University of Freiburg, Freiburg, Germany.

Abdullah Ely (A)

Wits/SAMRC Antiviral Gene Therapy Research Unit, School of Pathology, Faculty of Health Science, University of the Witwatersrand, Private Bag 3, WITS, Johannesburg, 2050, South Africa.

Patrick Arbuthnot (P)

Wits/SAMRC Antiviral Gene Therapy Research Unit, School of Pathology, Faculty of Health Science, University of the Witwatersrand, Private Bag 3, WITS, Johannesburg, 2050, South Africa. Patrick.Arbuthnot@wits.ac.za.

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Classifications MeSH