Prospects of viral vector-mediated delivery of sequences encoding anti-HBV designer endonucleases.
Journal
Gene therapy
ISSN: 1476-5462
Titre abrégé: Gene Ther
Pays: England
ID NLM: 9421525
Informations de publication
Date de publication:
24 May 2022
24 May 2022
Historique:
received:
01
12
2021
accepted:
06
05
2022
revised:
05
05
2022
pubmed:
24
5
2022
medline:
24
5
2022
entrez:
23
5
2022
Statut:
aheadofprint
Résumé
Available treatment for chronic hepatitis B virus (HBV) infection offers modest functional curative efficacy. The viral replicative intermediate comprising covalently closed circular DNA (cccDNA) is responsible for persistent chronic HBV infection. Hence, current efforts have focused on developing therapies that disable cccDNA. Employing gene editing tools has emerged as an attractive strategy, with the end goal of establishing permanently inactivated cccDNA. Although anti-HBV designer nucleases are effective in vivo, none has yet progressed to clinical trial. Lack of safe and efficient delivery systems remains the limiting factor. Several vectors may be used to deliver anti-HBV gene editor-encoding sequences, with viral vectors being at the forefront. Despite the challenges associated with packaging large gene editor-encoding sequences into viral vectors, advancement in the field is overcoming such limitations. Translation of viral vector-mediated gene editing against HBV to clinical application is within reach. This review discusses the prospects of delivering HBV targeted designer nucleases using viral vectors.
Identifiants
pubmed: 35606493
doi: 10.1038/s41434-022-00342-5
pii: 10.1038/s41434-022-00342-5
doi:
Types de publication
Journal Article
Review
Langues
eng
Sous-ensembles de citation
IM
Subventions
Organisme : National Research Foundation (NRF)
ID : Student bursary
Informations de copyright
© 2022. The Author(s), under exclusive licence to Springer Nature Limited.
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