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Related Experiment Video

Updated: Dec 28, 2025

Genome Editing in Mammalian Cell Lines using CRISPR-Cas
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CRISPR/Cas9 for genome editing: progress, implications and challenges.

Feng Zhang1, Yan Wen1, Xiong Guo2

  • 1Key Laboratory of Environment and Gene Related Diseases of Ministry Education, Faculty of Public Health, College of Medicine, Xi'an Jiaotong University, Xi'an, PR China.

Human Molecular Genetics
|March 22, 2014
PubMed
Summary
This summary is machine-generated.

The Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR)/CRISPR-associated (Cas) protein 9 system is a powerful genome editing tool. This review covers its mechanism, applications, challenges, and future therapeutic potential.

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Area of Science:

  • Molecular Biology
  • Genetics
  • Biotechnology

Background:

  • The Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR)/CRISPR-associated (Cas) protein 9 system is a revolutionary genome editing technology.
  • It enables precise manipulation of genomic elements for studying gene function in various biological contexts and diseases.

Purpose of the Study:

  • To review the molecular mechanisms underlying CRISPR/Cas9 genome editing.
  • To explore the diverse applications and inherent challenges of this technology.
  • To discuss the potential clinical therapeutic applications of CRISPR/Cas9 in the future.

Main Methods:

  • The CRISPR/Cas9 system utilizes a Cas9 nuclease guided by CRISPR RNA (crRNA) to induce targeted DNA double-strand breaks.
  • Cellular DNA repair pathways subsequently introduce insertions, deletions, or substitutions at the cleavage site.
  • Specificity is determined by crRNA sequence complementarity and the presence of a protospacer adjacent motif.

Main Results:

  • CRISPR/Cas9 offers a robust and multiplexable approach for precise genome editing.
  • The system's efficacy relies on accurate target sequence recognition and DNA repair processes.
  • Understanding these mechanisms is crucial for optimizing its application and safety.

Conclusions:

  • CRISPR/Cas9 is a versatile tool with significant implications for biological research and disease understanding.
  • Ongoing research addresses challenges related to specificity, delivery, and off-target effects.
  • The technology holds immense promise for future clinical therapeutics.