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CRISPR/Cas9 用于乙型肝炎病毒感染的治疗。

CRISPR/Cas9 for hepatitis B virus infection treatment.

机构信息

Center of Medical Genetics, Northwest Women's and Children's Hospital, Xi'an, Shaanxi, PR. China.

Center for Translational Medicine, The First Affiliated Hospital of Xi'an Jiaotong University, Xi'an, Shaanxi, PR. China.

出版信息

Immun Inflamm Dis. 2023 May;11(5):e866. doi: 10.1002/iid3.866.

Abstract

Hepatitis B virus (HBV) infection remains a global health challenge. Despite the availability of effective preventive vaccines, millions of people are at risk of cirrhosis and hepatocellular carcinoma. Current drug therapies inhibit viral replication, slow the progression of liver fibrosis and reduce infectivity, but they rarely remove the covalently sealed circular DNA (cccDNA) of the virus that causes HBV persistence. Alternative treatment strategies, including those based on CRISPR/cas9 knockout virus gene, can effectively inhibit HBV replication, so it has a good prospect. During chronic infection, some virus gene knockouts based on CRISPR/cas9 may even lead to cccDNA inactivation. This paper reviews the progress of different HBV CRISPR/cas9, vectors for delivering to the liver, and the current situation of preclinical and clinical research.

摘要

乙型肝炎病毒 (HBV) 感染仍然是一个全球性的健康挑战。尽管有有效的预防性疫苗,但仍有数百万人面临肝硬化和肝细胞癌的风险。目前的药物治疗方法可以抑制病毒复制、减缓肝纤维化的进展并降低传染性,但它们很少能消除导致乙型肝炎持续存在的病毒共价封闭环状 DNA (cccDNA)。替代治疗策略,包括基于 CRISPR/cas9 的病毒基因敲除,可有效抑制 HBV 复制,因此具有良好的前景。在慢性感染期间,基于 CRISPR/cas9 的一些病毒基因敲除甚至可能导致 cccDNA 失活。本文综述了不同的 HBV CRISPR/cas9、递送至肝脏的载体的研究进展,以及临床前和临床研究的现状。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/eae8/10170306/ca81ec0640fd/IID3-11-e866-g001.jpg

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