Related Experiment Video

Updated: Jul 27, 2025

Functional Assessment of BRCA1 variants using CRISPR-Mediated Base Editors
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Functional Assessment of BRCA1 variants using CRISPR-Mediated Base Editors

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CRISPR-Mediated Base Editing: Promises and Challenges for a Viable Oncotherapy Strategy

Lu Huang1,2, Chao Yang3, Yan Chen1

  • 1Department of Pharmacy, Sichuan Clinical Research Center for Cancer, Sichuan Cancer Center, Sichuan Cancer Hospital and Institute, Affiliated Cancer Hospital of University of Electronic Science and Technology of China, Chengdu, China.

Human Gene Therapy
|June 5, 2023
PubMed

Insights

Base editing, a CRISPR/Cas9 advancement, precisely modifies DNA/RNA without double-strand breaks. This technology offers new hope for treating genetic diseases and cancer by correcting point mutations.

Area of Science:

  • Molecular Biology
  • Genetics
  • Biotechnology

Background:

  • CRISPR/Cas9 technology enables precise genome editing.
  • Base editing is a derivative of CRISPR/Cas9, facilitating targeted single-base substitutions.
  • Point mutations are implicated in a significant portion of human genetic diseases and cancer development.

Purpose of the Study:

  • To review the main base editing technologies.
  • To discuss the applications and prospects of base editing in tumor research and therapy.
  • To elaborate on the delivery methods for base editors.

Main Methods:

  • Review of existing literature on base editing technologies.
  • Analysis of CRISPR/Cas9 system modifications for base editing.
  • Examination of studies on base editor applications in oncology.

Main Results:

  • Base editors enable precise single-base substitutions without inducing double-strand breaks.
  • Continuous improvements have enhanced base editor efficiency, specificity, and product purity.
  • Base editing shows significant promise for the study and treatment of genetic diseases and cancers.

Conclusions:

  • Base editing represents a significant advancement in genome editing, offering precise control over single-nucleotide changes.
  • The technology holds considerable potential for developing novel therapeutic strategies for genetic disorders and oncological conditions.
  • Further development and optimization of delivery methods are crucial for realizing the full clinical potential of base editors.
Keywords:
SNVsbase editingdelivery methodgene therapytumor

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