先进的基因治疗可治疗乙型肝炎病毒的慢性感染的最新进展

StephenW

Curr Opin HIV AIDS. 2020 Mar 5. doi: 10.1097/COH.0000000000000623. [Epub ahead of print]
Recent developments with advancing gene therapy to treat chronic infection with hepatitis B virus.
Maepa MB1, Jacobs R, van den Berg F, Arbuthnot P.
Author information

1
    Wits/SAMRC Antiviral Gene Therapy Research Unit, School of Pathology, Health Sciences Faculty, University of the Witwatersrand, Johannesburg, South Africa.

Abstract
PURPOSE OF REVIEW:

The available vaccine and therapies against hepatitis B virus (HBV) rarely eliminate chronic infection with the virus. High mortality resulting from complicating cirrhosis and hepatocellular carcinoma makes improving anti-HBV therapy an important priority. Recent advances with using gene therapy to counter HBV have potential and are the focus of this review.
RECENT FINDINGS:

The stable replication-competent HBV intermediate comprising covalently closed circular DNA (cccDNA) is the template for expression of all viral genes. Inactivating cccDNA has thus been a focus of research aimed at achieving cure for HBV infection. Many studies have reported profound inhibition of replication of the virus using silencing and editing techniques. Therapeutic gene silencing with synthetic short interfering RNA is now in clinical trials. Ability to mutate and permanently inactivate cccDNA with engineered gene editors, such as those derived from CRISPR/Cas or TALENs, is particularly appealing but has not yet reached clinical evaluation.
SUMMARY:

Gene silencing and gene editing potentially provide the means to cure HBV infection. However, achieving efficient delivery of therapeutic sequences, ensuring their specificity of action and progress with other antiviral strategies are likely to determine utility of gene therapy for chronic HBV infection.

PMID:
    32141890
DOI:
    10.1097/COH.0000000000000623

StephenW

Curr Opin HIV艾滋病。 2020年3月5日。doi：10.1097 / COH.0000000000000623。 [Epub提前发行]
先进的基因治疗可治疗乙型肝炎病毒的慢性感染的最新进展。
Maepa MB1，Jacobs R，van den Berg F，Arbuthnot P.
作者信息

1个
Wits / SAMRC抗病毒基因治疗研究组，南非约翰内斯堡威特沃特斯兰德大学健康科学学院病理学系。

抽象
审查目的：

现有的针对乙型肝炎病毒（HBV）的疫苗和疗法很少消除该病毒的慢性感染。复杂性肝硬化和肝细胞癌导致的高死亡率使改善抗-HBV治疗成为重要的优先事项。使用基因疗法对抗HBV的最新进展具有潜力，并且是本综述的重点。
最近发现：

包含共价闭合环状DNA（cccDNA）的稳定的具有复制能力的HBV中间体是表达所有病毒基因的模板。因此，灭活cccDNA是旨在治愈HBV感染的研究重点。许多研究报道了使用沉默和编辑技术对病毒复制的深刻抑制。合成短干扰RNA使治疗基因沉默目前正在临床试验中。利用工程化的基因编辑器（例如从CRISPR / Cas或TALENs衍生的基因编辑器）突变和永久灭活cccDNA的能力特别吸引人，但尚未达到临床评估。
摘要：

基因沉默和基因编辑可能提供治愈HBV感染的手段。但是，实现治疗序列的有效传递，确保其作用的特异性和其他抗病毒策略的进展可能决定基因治疗对慢性HBV感染的效用。

PMID：
32141890
DOI：
10.1097 / COH.0000000000000623

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