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Methods for CRISPR-Cas as Ribonucleoprotein Complex Delivery In Vivo
Alesya G Bykonya1, Alexander V Lavrov2, Svetlana A Smirnikhina2
1Sechenov University (MSMU), Trubetskaya str., 8, bldg. 2, Moscow, Russia, 119991. a.bykonya@gmail.com.
Molecular Biotechnology
|March 24, 2022
Summary
Efficiently delivering CRISPR-Cas9 components via ribonucleoprotein (RNP) complexes is crucial for in vivo genome editing. This review examines current methods and challenges in delivering these complexes to somatic cells for enhanced therapeutic potential.
Area of Science:
- Molecular Biology
- Gene Editing Technologies
- Biotechnology
Background:
- Efficient delivery of CRISPR-Cas components remains a significant challenge in genome editing.
- CRISPR-Cas delivery as Cas protein + single-guide RNA (sgRNA) ribonucleoprotein (RNP) complexes enhances on-target activity and reduces off-target effects.
Purpose of the Study:
- To review current scientific approaches for solving the problem of efficient in vivo delivery of CRISPR-Cas components.
- To explore various in vivo delivery methods for Cas9/sgRNA RNPs.
Main Methods:
- Review of existing scientific literature on in vivo CRISPR-Cas delivery.
- Analysis of different strategies for delivering Cas9/sgRNA RNP complexes into somatic cells.
Main Results:
- CRISPR-Cas RNP complexes offer improved specificity and efficacy compared to other delivery methods.
- Challenges include controlling nuclease dosage and achieving efficient delivery to target somatic cells in vivo.
- Various delivery systems are being developed to overcome these limitations.
Conclusions:
- Optimizing the delivery of CRISPR-Cas9 RNP complexes is essential for advancing in vivo genome editing applications.
- Further research into novel delivery methods is required to improve efficiency and control for therapeutic use.
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