Related Experiment Video
Updated: Jan 23, 2026
09:23
Delivery of the Cas9/sgRNA Ribonucleoprotein Complex in Immortalized and Primary Cells via Virus-like Particles "Nanoblades"
Published on: March 31, 2021
5.6K
Cas9 Ribonucleoprotein Complex Delivery: Methods and Applications for Neuroinflammation.
Lee A Campbell1, Christopher T Richie2, Nishad S Maggirwar2
1Intramural Research Program, Biomedical Research Center, National Institute on Drug Abuse, Suite 200, 251 Bayview Blvd, Baltimore, MD, 21224, USA. lee.campbell@nih.gov.
Summary
CRISPR/Cas9 gene editing advances human therapies by using ribonucleoprotein (RNP) complexes to minimize off-target mutations and immunogenicity. This approach enhances safety and efficacy for treating diseases like neuroinflammation.
Area of Science:
- Molecular Biology
- Gene Editing Technologies
- Neuroscience
Background:
- The CRISPR/Cas9 system offers powerful gene editing capabilities but faces challenges in human therapies, including off-target mutations and immunogenicity.
- Minimizing CRISPR/Cas9 component presence and temporal activity is crucial for therapeutic safety and efficacy.
- Pre-formed Cas9-gRNA ribonucleoprotein (RNP) complexes represent a promising strategy to achieve tighter temporal control.
Purpose of the Study:
- To review molecular modifications enabled by CRISPR/Cas9.
- To provide an overview of current methods for delivering Cas9 RNP complexes in vitro and in vivo.
- To explore the potential of Cas9 RNP delivery for targeting neuroinflammatory processes.
Main Methods:
- Discussion of CRISPR/Cas9 molecular modifications.
- Overview of in vitro and in vivo delivery methods for Cas9 RNP complexes.
- Review of applications in neuroinflammation, viral CNS infections, and neurodegenerative diseases.
Main Results:
- Cas9 RNP complexes offer a method to reduce the duration of Cas9 endonuclease activity, thereby mitigating potential off-target effects.
- Various delivery strategies for Cas9 RNP complexes have been developed for both experimental and potential therapeutic applications.
- Cas9 RNP delivery shows promise for addressing neuroinflammatory conditions.
Conclusions:
- Cas9 RNP delivery is a viable strategy for advancing CRISPR/Cas9 applications in both research and disease treatment.
- This approach holds significant potential for managing neuroinflammatory processes, including those associated with viral CNS infections and neurodegenerative diseases.
- Further development and application of Cas9 RNP delivery systems are warranted for safe and effective gene editing therapies.
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