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Related Concept Videos

CRISPR01:59

CRISPR

50.6K
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

17.0K
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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Related Experiment Video

Updated: Jun 25, 2025

Delivery of the Cas9/sgRNA Ribonucleoprotein Complex in Immortalized and Primary Cells via Virus-like Particles "Nanoblades"
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Delivery of the Cas9/sgRNA Ribonucleoprotein Complex in Immortalized and Primary Cells via Virus-like Particles "Nanoblades"

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Functionalized extracellular nanovesicles as advanced CRISPR delivery systems.

Siqing Wang1, Huimin Kong1, Chenya Zhuo1

  • 1Laboratory of Biomaterials and Translational Medicine, Center for Nanomedicine, The Third Affiliated Hospital, Sun Yat-sen University, Guangzhou 510630, China. zhuochy5@mail.sysu.edu.cn.

Biomaterials Science
|May 29, 2024
PubMed
Summary
This summary is machine-generated.

Extracellular nanovesicles (EVs) offer a promising solution for delivering CRISPR gene editing tools, overcoming current delivery challenges for genetic disease treatments. Research explores various functionalized EVs and engineering techniques for improved clinical translation.

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Designing, Packaging, and Delivery of High Titer CRISPR Retro and Lentiviruses via Stereotaxic Injection
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A Protocol for the Production of Integrase-deficient Lentiviral Vectors for CRISPR/Cas9-mediated Gene Knockout in Dividing Cells
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Area of Science:

  • Biotechnology
  • Nanomedicine
  • Gene Therapy

Background:

  • The Clustered Regularly Interspaced Short Palindromic Repeat (CRISPR) system shows great potential for treating genetic diseases.
  • Effective delivery strategies for CRISPR technology are crucial but currently underdeveloped for clinical applications.

Purpose of the Study:

  • To summarize functional extracellular nanovesicles (EVs) for CRISPR delivery.
  • To examine the intracellular pathways of various EV types.
  • To outline techniques for functionalizing EVs and discuss their clinical translation.

Main Methods:

  • Review of different types of functional EVs for CRISPR delivery (unmodified, modified, engineered virus-like particles, exosome-liposome hybrids).
  • Examination of distinct intracellular pathways associated with these EVs.
  • Outline of cutting-edge techniques for EV functionalization (producer cell, vesicle, and virus-like particle engineering).

Main Results:

  • Functional EVs present advantages like high biocompatibility, biological permeability, and low immunogenicity.
  • Various EV engineering techniques enable diverse CRISPR delivery capabilities.
  • Identified challenges and proposed design strategies for clinical translation of functionalized EVs.

Conclusions:

  • Functionalized extracellular nanovesicles are a promising platform for CRISPR delivery.
  • Further development and rational design are needed for successful clinical translation of EV-based CRISPR therapies.