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Related Concept Videos

CRISPR01:59

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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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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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What is Genetic Engineering?00:49

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Mouse Genome Engineering Using Designer Nucleases
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Targeting Hepatitis B Virus DNA Using Designer Gene Editors.

Henrik Zhang1, Thomas Tu1

  • 1Westmead Institute for Medical Research, University of Sydney School of Medicine and Health, 176 Hawkesbury Road, Westmead, NSW 2145, Australia.

Clinics in Liver Disease
|October 1, 2023
PubMed
Summary

Gene editing offers a potential cure for chronic hepatitis B virus (HBV) infection by disrupting viral DNA. While promising for HBV DNA inactivation and degradation, challenges in efficacy and safety require further solutions.

Keywords:
CRISPR-Cas9Hepatitis B virusIntegrated DNATALENZFNcccDNA

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Area of Science:

  • Hepatology
  • Virology
  • Gene Therapy

Background:

  • Chronic hepatitis B virus (HBV) infection lacks a definitive cure.
  • Key HBV forms, covalently closed circular DNA and integrated DNA, contribute to disease persistence, immune evasion, and cancer.
  • Current therapies do not effectively target these persistent HBV DNA forms.

Purpose of the Study:

  • To explore the potential of gene editing technologies for disrupting HBV DNA.
  • To evaluate the efficacy of gene editor-induced double-stranded breaks in targeting HBV genome.
  • To identify challenges and necessary solutions for safe and effective HBV gene editing therapies.

Main Methods:

  • Utilizing gene editing tools to create targeted double-stranded breaks within the HBV genome.
  • Investigating the impact of these breaks on HBV DNA inactivation and degradation.
  • Assessing the efficacy and safety profiles of the gene editing approach.

Main Results:

  • Gene editor-induced double-stranded breaks can lead to HBV gene inactivation.
  • Complete degradation of the HBV genome is achievable through precise gene editing.
  • The study highlights the potential of gene editing to disrupt persistent HBV DNA forms.

Conclusions:

  • Gene editing technologies present a promising therapeutic strategy for chronic hepatitis B.
  • Further research is needed to address efficacy and safety challenges for clinical application.
  • Targeting HBV DNA with gene editing could offer a pathway towards a functional cure for hepatitis B.