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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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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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Homologous Recombination02:31

Homologous Recombination

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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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What is Genetic Engineering?00:49

What is Genetic Engineering?

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Overview
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Updated: Oct 29, 2025

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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Recent Advances in CRISPR/Cas9 Directed Base Editing.

Nan Liu1, Lifang Zhou1, Junyan Qu2

  • 1Laboratory of Biotherapy, National Key Laboratory of Biotherapy, Cancer Center, West China Hospital, Sichuan University, Renmin Nanlu 17, Chengdu 610041, Sichuan, China.

Current Gene Therapy
|July 6, 2021
PubMed
Summary
This summary is machine-generated.

CRISPR-based base editing precisely converts single DNA bases, offering powerful tools for studying genetic diseases and advancing clinical applications with high efficiency.

Keywords:
CRISPR based techniquesbase editing tools.genomeshuman genetic diseasespoint mutationstargeted base editing

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Efficient PAM-Less Base Editing for Zebrafish Modeling of Human Genetic Disease with zSpRY-ABE8e
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Efficient PAM-Less Base Editing for Zebrafish Modeling of Human Genetic Disease with zSpRY-ABE8e
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Area of Science:

  • Genetics
  • Molecular Biology
  • Biotechnology

Background:

  • CRISPR-based techniques have revolutionized genome engineering.
  • Targeted base editing is a precise method for genomic modification.
  • Many human genetic diseases stem from point mutations.

Purpose of the Study:

  • To review recent advancements in base editing tools.
  • To discuss the challenges and potential of base editing.
  • To highlight implications for genetic disease research and clinical applications.

Main Methods:

  • Review of CRISPR-based targeted base editing techniques.
  • Analysis of base conversion efficiencies (C to T, A to G).
  • Discussion of applications in human genetic disease studies.

Main Results:

  • Base editing enables irreversible, specific single DNA base conversions.
  • High efficiency of base editing tools demonstrated.
  • Significant promise for clinical applications shown.

Conclusions:

  • Base editing is a powerful technique for precise genomic editing.
  • It holds great potential for understanding and treating human genetic diseases.
  • Further development is crucial for realizing its full clinical promise.