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Related Concept Videos

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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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 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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Engineered Metal-Organic Frameworks for Targeted CRISPR/Cas9 Gene Editing.

Navid Rabiee1,2,3,4, Mohammad Rabiee5

  • 1Department of Basic Medical Science, School of Medicine, Tsinghua University, Beijing 100084, China.

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|April 17, 2025
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Summary
This summary is machine-generated.

Engineered metal-organic frameworks (MOFs) offer advanced delivery for CRISPR/Cas9 gene editing, improving precision and efficiency. These biocompatible nanoplatforms show promise for safer, targeted gene therapies in precision medicine.

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Area of Science:

  • Nanotechnology
  • Biomedical Engineering
  • Molecular Biology

Background:

  • CRISPR/Cas9 gene editing requires efficient and precise delivery systems for therapeutic use.
  • Nonviral vectors are crucial for safe and effective gene delivery, but often face limitations.
  • Metal-organic frameworks (MOFs) present a novel class of inorganic nanocarriers with tunable properties.

Purpose of the Study:

  • To review the design and application of MOF-based nanoplatforms for CRISPR/Cas9 delivery.
  • To highlight MOFs' potential in enhancing gene-editing precision and efficiency.
  • To discuss MOF-based systems as advanced nonviral therapeutic delivery vectors.

Main Methods:

  • Exploration of MOF design strategies, including stimuli-responsive linkers and bioactive ligands.
  • Analysis of MOF-based nanoplatforms for targeted delivery of CRISPR/Cas9 components.
  • Review of comparative studies evaluating MOF performance against conventional nonviral systems.
  • Discussion of intracellular trafficking and preclinical therapeutic potential.

Main Results:

  • MOFs demonstrate tunable porosity, high cargo capacity, and good biocompatibility.
  • MOF-based systems enable controlled release of CRISPR/Cas9 payloads at target sites.
  • MOFs exhibit superior stability, transfection efficiency, and reduced off-target effects compared to conventional vectors.
  • MOF delivery systems show potential in overcoming immunogenicity and cytotoxicity issues.

Conclusions:

  • MOF-based nanoplatforms represent a transformative approach for CRISPR/Cas9 delivery.
  • These systems offer enhanced precision, efficiency, and safety for gene-editing therapies.
  • MOFs pave the way for advanced nonviral therapeutic delivery systems in precision medicine.