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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
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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
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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.
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