Prospects of viral vector-mediated delivery of sequences encoding anti-HBV designer endonucleases

被引:4
作者
Jacobs, Ridhwaanah [1 ]
Singh, Prashika [1 ]
Smith, Tiffany [1 ]
Arbuthnot, Patrick [1 ]
Maepa, Mohube Betty [1 ]
机构
[1] Univ Witwatersrand, Fac Hlth Sci, Wits SAMRC Antiviral Gene Therapy Res Unit, Johannesburg, South Africa
基金
新加坡国家研究基金会; 英国医学研究理事会;
关键词
HEPATITIS-B-VIRUS; HELPER-DEPENDENT ADENOVIRUS; ZINC-FINGER NUCLEASES; REPLICATION IN-VIVO; HOMOLOGOUS RECOMBINATION; ADENOASSOCIATED VIRUS; HEMATOPOIETIC STEM; CRISPR/CAS9; SYSTEM; BINDING DOMAINS; DNA CLEAVAGE;
D O I
10.1038/s41434-022-00342-5
中图分类号
Q5 [生物化学]; Q7 [分子生物学];
学科分类号
071010 ; 081704 ;
摘要
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.
引用
收藏
页码:8 / 15
页数:8
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