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Related Concept Videos

CRISPR01:59

CRISPR

52.6K
Genome editing technologies allow scientists to modify an organism’s DNA via the addition, removal, or rearrangement of genetic material at specific genomic locations. These types of techniques could potentially be used to cure genetic disorders such as hemophilia and sickle cell anemia. One popular and widely used DNA-editing research tool that could lead to safe and effective cures for genetic disorders is the CRISPR-Cas9 system. CRISPR-Cas9 stands for Clustered Regularly Interspaced...
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CRISPR/Cas9 Genome Editing01:28

CRISPR/Cas9 Genome Editing

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The CRISPR-Cas system serves as a bacterial defense mechanism against invading genetic elements such as viruses and plasmids, forming the foundation for its adaptation as a powerful genome-editing tool. Originally discovered in prokaryotes, this system has been repurposed to revolutionize genetic engineering across a wide range of organisms, including plants, animals, and humans. The core component, Cas9, is an endonuclease derived from Streptococcus pyogenes, capable of introducing...
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Updated: Aug 16, 2025

Introducing Point Mutations into Human Pluripotent Stem Cells Using Seamless Genome Editing
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Gene Editing Technologies to Target HBV cccDNA.

Maria Guadalupe Martinez1, Elena Smekalova2, Emmanuel Combe1

  • 1INSERM U1052, CNRS UMR-5286, Cancer Research Center of Lyon (CRCL), 69008 Lyon, France.

Viruses
|December 23, 2022
PubMed
Summary

Gene editing technologies offer new hope for treating chronic Hepatitis B virus (HBV) infection by targeting the persistent viral DNA. These novel approaches aim to reduce or eliminate HBV covalently closed circular DNA (cccDNA), preventing viral rebound.

Keywords:
CRISPR/CasHBVTALENZNFbase editingcccDNAgene editinghepatitis B virusmeganuclease

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Area of Science:

  • Hepatology and Virology
  • Molecular Biology
  • Gene Therapy

Background:

  • Chronic Hepatitis B virus (HBV) infection is a major global health concern, leading to cirrhosis and liver cancer.
  • Current antiviral therapies suppress HBV replication but fail to eliminate intrahepatic covalently closed circular DNA (cccDNA), causing viral persistence.
  • Life-long treatment is necessary to manage chronic HBV due to the presence of cccDNA, necessitating novel therapeutic strategies.

Purpose of the Study:

  • To review the latest advancements in gene editing and related technologies for targeting HBV DNA.
  • To explore the potential of these approaches in reducing, silencing, or eradicating HBV cccDNA in infected hepatocytes.
  • To address the need for curative therapies that prevent HBV reemergence after treatment cessation.

Main Methods:

  • Review of current literature on gene editing tools (e.g., CRISPR-Cas) and their application to HBV.
  • Analysis of strategies for delivering gene editing components to hepatocytes.
  • Discussion of methods to assess the efficacy of gene editing in targeting HBV cccDNA.

Main Results:

  • Gene editing technologies show promise in directly targeting and modifying HBV DNA, including cccDNA.
  • Successful reduction or silencing of cccDNA has been demonstrated in preclinical models.
  • These approaches offer a potential pathway to a functional cure for chronic HBV infection.

Conclusions:

  • Gene editing represents a promising therapeutic strategy for chronic HBV infection.
  • Targeting HBV cccDNA with gene editing could lead to a functional cure, eliminating the need for long-term treatment.
  • Further research and clinical development are crucial to translate these findings into effective therapies.