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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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CRISPR/Cas9 Genome Editing01:28

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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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CRISPR and crRNAs02:53

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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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Homologous Recombination02:31

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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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Updated: Dec 15, 2025

Genome Editing in Mammalian Cell Lines using CRISPR-Cas
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Progress and challenges towards CRISPR/Cas clinical translation.

Daniel Rosenblum1, Anna Gutkin1, Niels Dammes1

  • 1Laboratory of Precision NanoMedicine, School of Molecular Cell Biology and Biotechnology, George S. Wise Faculty of Life Sciences, Tel Aviv University, Tel-Aviv, Israel; Department of Materials Sciences and Engineering, Iby and Aladar Fleischman Faculty of Engineering, Tel Aviv University, Tel Aviv, Israel; Center for Nanoscience and Nanotechnology, Tel Aviv University, Tel Aviv, Israel; Cancer Biology Research Center, Tel Aviv University, Tel Aviv, Israel.

Advanced Drug Delivery Reviews
|July 14, 2020
PubMed
Summary

CRISPR gene editing offers powerful genome manipulation but faces challenges like off-target effects and delivery. Ongoing research focuses on improving Cas nucleases and developing safe in vivo delivery methods for clinical applications.

Keywords:
CRISPR/Cas systemsCancer immunotherapyChallengesClinical translationDeliveryGene therapyNon-viral vectorsViral vectors

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Area of Science:

  • Molecular Biology
  • Genetics
  • Biotechnology

Background:

  • CRISPR/Cas systems provide advanced genome editing capabilities for research and therapeutic applications.
  • Clinical translation of CRISPR technology is limited by challenges including off-target mutations, low editing efficiency, and immunogenicity.
  • Efficient and safe delivery of CRISPR components in vivo remains a significant hurdle for therapeutic development.

Purpose of the Study:

  • To review the current CRISPR/Cas toolbox for genome editing.
  • To summarize novel in vivo delivery vehicles for CRISPR/Cas systems.
  • To discuss challenges and highlight clinical applications of CRISPR technology.

Main Methods:

  • Literature review of CRISPR/Cas systems and genome editing techniques.
  • Analysis of viral and non-viral delivery strategies for in vivo CRISPR applications.
  • Discussion of current clinical trials and future prospects.

Main Results:

  • An overview of engineered Cas nucleases with enhanced precision and efficacy.
  • A summary of various delivery systems, including viral vectors and lipid nanoparticles.
  • Identification of key challenges hindering clinical translation, such as delivery efficiency and immune response.

Conclusions:

  • CRISPR/Cas technology holds immense therapeutic potential, but overcoming delivery and safety challenges is crucial.
  • Continued development of precise nucleases and effective delivery vehicles will accelerate clinical translation.
  • Highlighting current clinical applications demonstrates the progressing impact of CRISPR in medicine.