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Related Concept Videos

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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Advances in Engineered Virus-Like Particles for Genome Editing and Therapy.

Se Hyeok Son1, Seeone Woo1, Ayeon Choi1

  • 1College of Pharmacy, Chung-Ang University, 84 Heukseok-ro, Dongjak-gu, Seoul, 06974, South Korea.

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Engineered virus-like particles (eVLPs) offer safe and efficient delivery of genome editing tools like CRISPR-Cas9. These advanced eVLPs show promise for treating genetic diseases and are moving toward clinical applications.

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Area of Science:

  • Biotechnology
  • Gene Therapy
  • Molecular Biology

Background:

  • Engineered virus-like particles (eVLPs) are emerging as advanced delivery systems for genome editing technologies.
  • They combine viral vector entry efficiency with nonviral platform safety for transient delivery of ribonucleoproteins (e.g., Cas9, base editors, prime editors).

Purpose of the Study:

  • To review the principles, advances, and therapeutic potential of eVLPs for genome editing applications.
  • To discuss the challenges and future directions for eVLP-based gene editing therapies.

Main Methods:

  • Summary of structural and production principles of eVLPs.
  • Survey of key developmental advances in eVLP design and engineering.
  • Analysis of pseudotyping strategies for cell-type-specific targeting.

Main Results:

  • Rational engineering and directed optimization have improved eVLP assembly, cargo stability, and editing efficiency.
  • Pseudotyping enhances eVLP versatility for targeted delivery.
  • Preclinical studies demonstrate eVLP potential in monogenic and complex disease models.

Conclusions:

  • eVLPs represent a promising platform for transient delivery of genome editing agents.
  • Advancements in eVLP technology support their progression toward clinical translation for various genetic disorders.
  • Further development is needed to address future challenges in therapeutic applications.