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Related Concept Videos

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Gene Editing by Extracellular Vesicles.

Dmitry Kostyushev1, Anastasiya Kostyusheva1, Sergey Brezgin1,2

  • 1National Medical Research Center of Tuberculosis and Infectious Diseases, Ministry of Health, Moscow 127994, Russia.

International Journal of Molecular Sciences
|October 8, 2020
PubMed
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Extracellular vesicles (EVs) offer a promising solution for delivering CRISPR/Cas gene editing tools. These natural nanoparticles provide a safe and effective method for targeted delivery of CRISPR/Cas ribonucleoprotein complexes.

Keywords:
biodistributionexosomesgene editingnanoblades, stem cells, mesenchymal stem cells.nanomedicinesnanoparticlesnanovesiclespharmacokinetics

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Area of Science:

  • Molecular Biology
  • Gene Editing Technologies
  • Nanomedicine

Background:

  • CRISPR/Cas technologies have advanced significantly, enabling precise genome and epigenome modification.
  • Current limitations in CRISPR/Cas application stem from the lack of efficient delivery systems.
  • Extracellular vesicles (EVs) are emerging as a viable delivery platform for gene editing components.

Purpose of the Study:

  • To review recent advancements in developing vehicles for CRISPR/Cas delivery.
  • To highlight the potential of extracellular vesicles (EVs) as a superior delivery system for CRISPR/Cas ribonucleoprotein complexes.
  • To discuss strategies for engineering EVs for enhanced targeting and cargo loading.

Main Methods:

  • Review of recent literature on CRISPR/Cas delivery systems.
  • Focus on extracellular vesicles (EVs) as a delivery vehicle.
  • Discussion of molecular tools for EV cargo loading and surface modification.

Main Results:

  • Extracellular vesicles (EVs) demonstrate key advantages including biocompatibility, safety, design flexibility, and barrier crossing capabilities.
  • Methods for controllable loading of CRISPR/Cas ribonucleoprotein complexes into EVs are available.
  • Surface engineering of EVs allows for targeted delivery to specific tissues using ligands, peptides, or nanobodies.

Conclusions:

  • Extracellular vesicles (EVs) represent a highly promising and versatile platform for the delivery of CRISPR/Cas systems.
  • EVs can be engineered for efficient and targeted delivery of CRISPR/Cas ribonucleoprotein complexes.
  • Both endogenous and exogenous loading strategies for EVs are feasible, expanding delivery options.