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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

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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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The Antiviral System of Bacteria and Archaea: CRISPR01:23

The Antiviral System of Bacteria and Archaea: CRISPR

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CRISPR stands for Clustered Regularly Interspaced Short Palindromic Repeats is a adaptive immune system found in bacteria and archaea that protects against viral infections. This system enables prokaryotic cells to identify, remember, and neutralize foreign genetic elements, primarily bacteriophages, by storing fragments of the invader’s DNA as a genetic memory.The CRISPR immune response begins during an initial infection. Cas (CRISPR-associated) proteins play a central role in this...
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Updated: Jan 3, 2026

Genome Editing in Mammalian Cell Lines using CRISPR-Cas
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CRISPR/Cas system: An emerging technology in stem cell research.

Maria Teresa Valenti1, Michela Serena2, Luca Dalle Carbonare3

  • 1Department of Medicine, Section of Internal Medicine D, University of Verona, Verona 37134, Italy. mariateresa.valenti@univr.it.

World Journal of Stem Cells
|November 27, 2019
PubMed
Summary

CRISPR/Cas9 technology revolutionizes genome engineering for stem cell research. This review explores its applications in degenerative diseases, highlighting therapeutic potential and future challenges in medical sciences.

Keywords:
CRISPR/Cas9Degenerative diseasesGene editingStem cells

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Area of Science:

  • Genetics and Molecular Biology
  • Regenerative Medicine
  • Biotechnology

Background:

  • Genome modification technologies have evolved significantly since DNA's discovery.
  • Precise gene editing is crucial for understanding gene function and developing therapies.
  • Clustered regularly interspaced short palindromic repeats (CRISPR)/Cas9 has emerged as a revolutionary genome engineering tool.

Purpose of the Study:

  • To review the applications of CRISPR/Cas9 technology in stem cell research for degenerative diseases.
  • To explore the potential of CRISPR/Cas9 in developing new therapeutic strategies.
  • To discuss the challenges and future perspectives of CRISPR/Cas9 in medical sciences.

Main Methods:

  • Literature review of CRISPR/Cas9 applications in stem cell research.
  • Analysis of studies focusing on degenerative diseases and CRISPR/Cas9 interventions.
  • Discussion of technological advancements and clinical translation challenges.

Main Results:

  • CRISPR/Cas9 enables precise gene modification in stem cells for disease modeling.
  • Applications include correcting genetic mutations and engineering cells for therapeutic purposes.
  • The technology shows promise for developing novel treatments for degenerative conditions.

Conclusions:

  • CRISPR/Cas9 is a powerful tool for advancing stem cell research and regenerative medicine.
  • Its application in degenerative diseases offers significant therapeutic potential.
  • Overcoming current challenges will be key to its successful translation into clinical practice.