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Related Concept Videos

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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Conservative Site-specific Recombination and Phase Variation02:53

Conservative Site-specific Recombination and Phase Variation

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Because the DNA segments are cut and reorganized in a direction-specific manner, site-specific recombination has emerged as an efficient genetic engineering technique. Flippase and Cyclization recombinases or Flp and Cre, respectively, are two members of the tyrosine recombinase family derived from bacteriophages, that are used to mediate site-specific DNA insertions, deletions, and targeted expression of proteins in mammalian cell lines.
The recognition sites for Cre recombinase called LoxP...
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CRISPR01:59

CRISPR

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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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CRISPR and crRNAs02:53

CRISPR and crRNAs

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Bacteria and archaea are susceptible to viral infections just like eukaryotes; therefore, they have developed a unique adaptive immune system to protect themselves. Clustered regularly interspaced short palindromic repeats and CRISPR-associated proteins (CRISPR-Cas) are present in more than 45% of known bacteria and 90% of known archaea.
The CRISPR-Cas system stores a copy of foreign DNA in the host genome and uses it to identify the foreign DNA upon reinfection. CRISPR-Cas has three different...
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Base Excision Repair01:54

Base Excision Repair

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One of the common DNA damages is the chemical alteration of single bases by alkylation, oxidation, or deamination. The altered bases cause mispairing and strand breakage during replication. This type of damage causes minimal change to the DNA double helix structure and can be repaired by the base excision repair (BER) pathways. BER corrects damaged DNA sequences by removing the damaged base and restoring the original base sequence using the complementary strand as a template.
The first step of...
22.5K
Homologous Recombination02:31

Homologous Recombination

50.6K
The basic reaction of homologous recombination (HR) involves two chromatids that contain DNA sequences sharing a significant stretch of identity. One of these sequences uses a strand from another as a template to synthesize DNA in an enzyme-catalyzed reaction. The final product is a novel amalgamation of the two substrates. To ensure an accurate recombination of sequences, HR is restricted to the S and G2 phases of the cell cycle. At these stages, the DNA has been replicated already and the...
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Updated: Jul 16, 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

Published on: February 28, 2021

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Precise genome editing with base editors.

Hongcai Liu1,2, Yao Zhu1,2, Minjie Li1,2

  • 1Institute of Systems Medicine, Chinese Academy of Medical Sciences & Peking Union Medical College, Beijing, China.

Medical Review (2021)
|September 19, 2023
PubMed
Summary
This summary is machine-generated.

Genome editing technologies like base editing and prime editing offer precise ways to correct genetic mutations without causing DNA breaks. These advancements hold significant therapeutic potential for treating genetic disorders.

Keywords:
adenine base editorbase editingclustered regularly interspaced short palindromic repeats (CRISPR)/ CRISPR-associated proteincytosine base editor

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Enhanced Genome Editing with Cas9 Ribonucleoprotein in Diverse Cells and Organisms
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Efficient PAM-Less Base Editing for Zebrafish Modeling of Human Genetic Disease with zSpRY-ABE8e
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Enhanced Genome Editing with Cas9 Ribonucleoprotein in Diverse Cells and Organisms
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Enhanced Genome Editing with Cas9 Ribonucleoprotein in Diverse Cells and Organisms

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Area of Science:

  • Genetics
  • Molecular Biology
  • Biotechnology

Background:

  • Single-nucleotide variants are a major cause of human genetic diseases.
  • Genome editing offers therapeutic potential by correcting pathogenic point mutations.
  • Conventional gene editing methods can cause double-stranded DNA breaks (DSBs).

Purpose of the Study:

  • To review the evolution of base editors (BEs).
  • To discuss the limitations of current base editing technologies.
  • To explore the future perspectives of base editing for therapeutic strategies.

Main Methods:

  • Literature review of base editing technologies.
  • Analysis of base editor efficiency and accuracy.
  • Discussion of therapeutic applications and challenges.

Main Results:

  • Base editing and prime editing enable precise nucleotide conversion without DSBs.
  • A variety of base editors have been developed for diverse applications.
  • These technologies show promise for treating genetic disorders.

Conclusions:

  • Base editing represents a significant advancement in genome editing for therapeutic purposes.
  • Overcoming current limitations is crucial for realizing the full potential of base editing.
  • Future research should focus on improving base editor safety, efficiency, and delivery for clinical applications.