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Related Concept Videos

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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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.
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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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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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Related Experiment Video

Updated: Feb 23, 2026

Efficient Genome Editing of Mice by CRISPR Electroporation of Zygotes
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CRISPR: Established Editor of Human Embryos?

Xiao-Jiang Li1, Zhuchi Tu2, Weili Yang2

  • 1Ministry of Education CNS Regeneration Collaborative Joint Laboratory, Guangdong-Hongkong-Macau Institute of CNS Regeneration, Jinan University, Guangzhou, 510632, China; Department of Human Genetics, Emory University School of Medicine, Atlanta, GA 30322, USA.

Cell Stem Cell
|September 9, 2017
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Summary

CRISPR-Cas9 gene editing successfully repaired a genetic mutation in human embryos. This novel approach minimized mosaicism and off-target effects, advancing clinical applications for genetic diseases.

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Area of Science:

  • Genetics
  • Molecular Biology
  • Biotechnology

Background:

  • CRISPR-Cas9 gene editing holds promise for correcting genetic mutations.
  • Off-target mutations and mosaicism are significant challenges for clinical translation.
  • Efficient and precise gene correction in human embryos is crucial for therapeutic development.

Purpose of the Study:

  • To develop and evaluate a CRISPR-Cas9 strategy for precise gene correction in human embryos.
  • To assess the efficacy of the approach in repairing a specific genetic mutation.
  • To investigate the occurrence of off-target effects and mosaicism associated with the gene editing process.

Main Methods:

  • Utilized an elegant CRISPR-Cas9 system for targeted gene repair in human embryos.
  • Employed advanced molecular techniques to verify successful mutation correction.
  • Analyzed embryos for off-target mutations and mosaicism using sensitive detection methods.

Main Results:

  • The CRISPR-Cas9 approach effectively repaired the targeted genetic mutation in human embryos.
  • Negligible levels of mosaicism were observed post-editing.
  • No detectable off-target effects were identified, indicating high specificity.

Conclusions:

  • This study demonstrates a highly efficient and precise CRISPR-Cas9-mediated gene correction strategy for human embryos.
  • The developed method significantly overcomes major hurdles of off-target effects and mosaicism.
  • These findings pave the way for the clinical application of CRISPR-Cas9 in treating genetic disorders.