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DOI 10.1038/s41434-022-00342-5

Медицинская библиотека · Gene therapy · 2022

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

Jacobs Ridhwaanah, Singh Prashika, Smith Tiffany, Arbuthnot Patrick, Maepa Mohube Betty

СтатьяОбзорПоддержка исследования: вне правительства СШАПеревод в очереди

Аннотация

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.

Для цитирования: Jacobs Ridhwaanah, Singh Prashika, Smith Tiffany, Arbuthnot Patrick, Maepa Mohube Betty. Prospects of viral vector-mediated delivery of sequences encoding anti-HBV designer endonucleases.. Gene therapy. 2022. DOI: 10.1038/s41434-022-00342-5.

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