Related Experiment Video
Updated: Aug 25, 2025
09:51
Enhanced Genome Editing with Cas9 Ribonucleoprotein in Diverse Cells and Organisms
Published on: May 25, 2018
34.2K
Advances in CRISPR Delivery Methods: Perspectives and Challenges.
Selami Demirci1, Khaled Essawi1,2, Paula Germino-Watnick1
1Cellular and Molecular Therapeutics Branch, National Heart Lung and Blood Institutes (NHLBI), National Institutes of Health (NIH), Bethesda, Maryland, USA; and College of Applied Medical Sciences, Jazan University, Jazan, Saudi Arabia.
The CRISPR Journal
|October 19, 2022
Summary
Genome editing offers permanent disease correction, with CRISPR-Cas technology being highly efficient. Effective delivery of gene editing tools to target cells is crucial for therapeutic success.
Area of Science:
- Biotechnology
- Molecular Biology
- Genetics
Background:
- Genome editing technologies promise permanent disease correction, moving beyond symptomatic treatments.
- Clustered regularly interspaced short palindromic repeats (CRISPR)-Cas systems are favored for their efficiency, simplicity, and cost-effectiveness.
Purpose of the Study:
- To review viral and nonviral delivery systems for CRISPR-Cas gene editing machinery.
- To highlight the importance of optimizing delivery methods alongside editing technologies.
Main Methods:
- Discussion of viral delivery systems.
- Analysis of nonviral delivery systems.
- Evaluation of delivery parameters including payload, immunogenicity, and efficacy.
Main Results:
- CRISPR-Cas technology is a leading gene editing tool.
- Delivery of gene editing machinery remains a critical challenge.
- Various viral and nonviral strategies are being developed and refined.
Conclusions:
- Simultaneous advancement of gene editing and delivery systems is essential.
- Optimized delivery is key to realizing the therapeutic potential of genome editing.
- Future directions involve improving safety, efficacy, and clinical application of delivery modalities.
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