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Updated: Jul 13, 2025

Designing, Packaging, and Delivery of High Titer CRISPR Retro and Lentiviruses via Stereotaxic Injection
Published on: May 23, 2016
CRISPR-Cas9 delivery strategies with engineered extracellular vesicles
Yaoyao Lu1, Kelly Godbout1, Gabriel Lamothe1
1Centre de Recherche du CHU de Québec -Université Laval, Québec city, QC G1V4G2, Canada.
Extracellular vesicles (EVs) show promise for delivering CRISPR-Cas9 gene editing tools. This review explores methods to enhance EV loading and targeting for effective genetic disease therapies.
Area of Science:
- Biotechnology
- Molecular Biology
- Genetics
Background:
- Therapeutic genome editing offers potential cures for genetic diseases by correcting mutations.
- CRISPR-Cas9 systems are advanced gene editing tools due to their efficiency and versatility.
- Safe and effective delivery of CRISPR-Cas9 to target organs remains a significant challenge in gene therapy.
Purpose of the Study:
- To review recent advancements in utilizing extracellular vesicles (EVs) for CRISPR-Cas9 delivery.
- To highlight strategies for enhancing EV loading, surface functionalization, and targeting efficiency.
- To discuss the potential of EV-mediated CRISPR-Cas9 for treating genetic diseases.
Main Methods:
- Review of current literature on extracellular vesicle (EV) biogenesis and function.
- Analysis of methods for loading CRISPR-Cas9 components into EVs.
- Examination of surface modification techniques to improve EV targeting.
- Evaluation of strategies to enhance CRISPR-Cas9 delivery efficiency to specific tissues and cells.
Main Results:
- Extracellular vesicles (EVs) can naturally transport therapeutic molecules, including CRISPR-Cas9 systems.
- Various loading techniques are being developed to encapsulate CRISPR-Cas9 within EVs.
- Surface functionalization of EVs can improve their specificity and uptake by target cells.
- Optimized EV delivery systems show potential for efficient in vivo gene editing.
Conclusions:
- Extracellular vesicles (EVs) represent a promising platform for the targeted delivery of CRISPR-Cas9 gene editing technology.
- Advancements in EV loading and functionalization are crucial for overcoming current delivery hurdles.
- EV-mediated CRISPR-Cas9 delivery holds significant potential for the future of genetic medicine and disease treatment.
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