Artificial Liver Center, Beijing Youan Hospital, Capital Medical University, Beijing 100069, P.R. China.
Department of Gastroenterology, Pennsylvania Hospital, University of Pennsylvania, Philadelphia, PA 19104, USA.
Mol Med Rep. 2017 Nov;16(5):7199-7204. doi: 10.3892/mmr.2017.7518. Epub 2017 Sep 19.
Current therapeutic strategies cannot eradicate hepatitis B virus covalently closed circular DNA (HBV cccDNA), which accounts for the persistence of HBV infection. Very recently, the clustered regularly interspaced short palindromic repeat (CRISPR)/CRISPR‑associated protein 9 (Cas9) system has been used as an efficient and powerful tool for viral genome editing. Given that the primary duck hepatocyte (PDH) infected with duck hepatitis B virus (DHBV) has been widely used to study human HBV infection in vitro, the present study aimed to demonstrate the targeted inhibition of DHBV DNA, especially cccDNA, by the CRISPR/Cas9 system using this model. We designed six single‑guide RNAs (sgRNA1‑6) targeting the DHBV genome. The sgRNA/Cas9 plasmid was transfected into DHBV‑infected PDHs, and then DHBV total DNA (in culture medium and PDHs) and cccDNA were quantified by reverse transcription‑quantitative polymerase chain reaction. The combined inhibition of CRISPR/Cas9 system and entecavir (ETV) was also assessed. Two sgRNAs, sgRNA4 and sgRNA6, exhibited efficient inhibition on DHBV total DNA (77.23 and 86.51%, respectively), cccDNA (75.67 and 85.34%, respectively) in PDHs, as well as DHBV total DNA in the culture medium (62.17 and 59.52%, respectively). The inhibition remained or enhanced from day 5 to day 9 following transfection. The combination of the CRISPR/Cas9 system and ETV further increased the inhibitory effect on DHBV total DNA in PDHs and culture medium, but not cccDNA. The CRISPR/Cas9 system has the potential to be a useful tool for the suppression of DHBV DNA.
目前的治疗策略无法消除共价闭合环状 DNA(HBV cccDNA),这是乙型肝炎病毒(HBV)感染持续存在的原因。最近,成簇规律间隔短回文重复(CRISPR)/CRISPR 相关蛋白 9(Cas9)系统已被用作病毒基因组编辑的有效和强大工具。鉴于原发性鸭肝细胞(PDH)感染鸭乙型肝炎病毒(DHBV)已被广泛用于体外研究人类 HBV 感染,本研究旨在使用该模型证明 CRISPR/Cas9 系统对 DHBV DNA,特别是 cccDNA 的靶向抑制作用。我们设计了六个针对 DHBV 基因组的单指导 RNA(sgRNA1-6)。sgRNA/Cas9 质粒转染到 DHBV 感染的 PDH 中,然后通过逆转录定量聚合酶链反应定量检测培养基和 PDH 中的 DHBV 总 DNA(cccDNA)和 cccDNA。还评估了 CRISPR/Cas9 系统和恩替卡韦(ETV)联合抑制的效果。两个 sgRNA,sgRNA4 和 sgRNA6,对 PDH 中的 DHBV 总 DNA(分别为 77.23%和 86.51%)和 cccDNA(分别为 75.67%和 85.34%),以及培养基中的 DHBV 总 DNA(分别为 62.17%和 59.52%)均具有高效抑制作用。转染后第 5 天至第 9 天,抑制作用保持或增强。CRISPR/Cas9 系统与 ETV 的联合应用进一步提高了 PDH 和培养基中 DHBV 总 DNA 的抑制作用,但对 cccDNA 没有影响。CRISPR/Cas9 系统有可能成为抑制 DHBV DNA 的有用工具。