首页> 外国专利> LIPID NANOPARTICLE (LNP)-MEDIATED DELIVERY OF A CRISPR-EXPRESSING PLASMID DNA FOR TREATING CHRONIC HEPATITIS B VIRUS INFECTION

LIPID NANOPARTICLE (LNP)-MEDIATED DELIVERY OF A CRISPR-EXPRESSING PLASMID DNA FOR TREATING CHRONIC HEPATITIS B VIRUS INFECTION

机译:脂质纳米粒子(LNP)介导的表达CRISPR的质粒DNA的治疗慢性乙型肝炎病毒感染的治疗

摘要

Provided herein is a CRISPR-expressing plasmid DNA and a gene delivery system targeted to hepatocytes infected with hepatitis B virus. Also provided are methods of directing CRISPR system formation in such cells to ensure enhanced specificity for target recognition to alter, improve, or treat chronic HBV infection. In addition the CpG content of the expressing plasmid DNA has been minimized to reduce inflammation and maximize gene expression.
机译:本文提供了表达CRISPR的质粒DNA和靶向被乙型肝炎病毒感染的肝细胞的基因递送系统。还提供了指导在此类细胞中形成CRISPR系统的方法,以确保增强对靶标识别的特异性,以改变,改善或治疗慢性HBV感染。另外,表达质粒DNA的CpG含量已被最小化以减少炎症并最大化基因表达。

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