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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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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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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.
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Conservative Site-specific Recombination and Phase Variation02:53

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Because the DNA segments are cut and reorganized in a direction-specific manner, site-specific recombination has emerged as an efficient genetic engineering technique. Flippase and Cyclization recombinases or Flp and Cre, respectively, are two members of the tyrosine recombinase family derived from bacteriophages, that are used to mediate site-specific DNA insertions, deletions, and targeted expression of proteins in mammalian cell lines.
The recognition sites for Cre recombinase called LoxP...
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Updated: May 15, 2025

Genome Editing in Mammalian Cell Lines using CRISPR-Cas
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Genome Editing in Mammalian Cell Lines using CRISPR-Cas

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Exploring Advanced CRISPR Delivery Technologies for Therapeutic Genome Editing.

Neda Rostami1, Mohammad Mahmoudi Gomari2, Edris Choupani3

  • 1Department of Chemical Engineering Arak University Arak 3848177584 Iran.

Small Science
|April 11, 2025
PubMed
Summary
This summary is machine-generated.

Engineered carriers enhance CRISPR genome editing delivery in vivo, overcoming challenges like low efficiency and off-target effects. These platforms improve stability and precision for therapeutic applications.

Keywords:
biomaterialsclustered regularly interspaced short palindromic repeatexosomesgenome editingnanoparticles

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Area of Science:

  • Biotechnology
  • Molecular Biology
  • Genetics

Background:

  • Genetic material governs organismal structure and function.
  • Genome manipulation offers potential for correcting abnormalities and introducing new traits.
  • CRISPR technology provides precise genome editing capabilities.

Purpose of the Study:

  • To review recent advances in CRISPR-based genome editing delivery systems.
  • To explore the benefits and challenges of various engineered carrier platforms.
  • To provide insights into the clinical utility and future prospects of CRISPR delivery technologies.

Main Methods:

  • Review of scientific literature on CRISPR delivery systems.
  • Examination of engineered carrier platforms, including nanocarriers, viral particles, and exosomes.
  • Analysis of challenges such as delivery efficiency, off-target effects, and stability.

Main Results:

  • Engineered carrier platforms significantly improve CRISPR delivery efficiency, stability, and precision.
  • These platforms mitigate off-target effects and reduce side effects associated with genome editing.
  • Various carriers, including nanocarriers, viral particles, and exosomes, show promise for in vivo applications.

Conclusions:

  • Engineered carriers are crucial for advancing CRISPR genome editing for therapeutic applications.
  • Further research into carrier platforms will enhance clinical utility and expand the scope of genome engineering.
  • Targeted and precise delivery systems are key to overcoming current limitations of CRISPR technology.