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Stimulus-Responsive Smart Nanoparticles-Based CRISPR-Cas Delivery for Therapeutic Genome Editing
Muhammad Naeem1, Mubasher Zahir Hoque1, Muhammad Ovais2
1Department of Bioengineering, King Fahd University of Petroleum and Minerals (KFUPM), Dhahran 31261, Saudi Arabia.
International Journal of Molecular Sciences
|October 23, 2021
Summary
Smart nanoparticles offer a novel solution for delivering CRISPR-Cas9 gene editing tools, overcoming delivery challenges and reducing off-target effects for advanced genome surgery applications.
Area of Science:
- Biotechnology
- Genomics
- Nanomedicine
Background:
- CRISPR-Cas technology revolutionizes genome editing for therapeutic development.
- Clinical application of CRISPR-Cas9 faces challenges in efficient delivery and off-target effects.
- Current viral and non-viral vectors have limitations including immune response and specificity.
Purpose of the Study:
- To review recent advances in stimulus-based smart nanoparticles for CRISPR/Cas9 delivery.
- To highlight the potential of smart nanoparticles in overcoming current delivery barriers.
- To provide information for the utilization of smart nanoparticles in biomedical applications and genome editing.
Main Methods:
- Review of recent scientific literature on smart nanoparticles for CRISPR/Cas9 delivery.
- Analysis of endogenous and exogenous stimulus-responsive nanoparticle systems.
- Evaluation of nanoparticle strategies for targeted delivery based on cellular environments.
Main Results:
- Smart nanoparticles can be designed to respond to various endogenous (pH, enzyme, redox, ATP) and exogenous (magnetic, ultrasound, temperature, light) stimuli.
- These stimulus-responsive systems offer improved specificity and efficiency in delivering CRISPR/Cas9 machinery.
- Leveraging cellular microenvironments and external triggers can minimize off-target effects.
Conclusions:
- Stimulus-based smart nanoparticles represent a promising approach for advancing CRISPR/Cas9 delivery.
- These advanced delivery systems hold potential for safer and more effective therapeutic genome editing.
- Further research is needed for cautious utilization in basic biomedical applications and clinical translation.
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