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Updated: Aug 8, 2025
09:51
Enhanced Genome Editing with Cas9 Ribonucleoprotein in Diverse Cells and Organisms
Published on: May 25, 2018
34.2K
New advances in CRISPR/Cas-mediated precise gene-editing techniques
Chris Richardson1, Robert N Kelsh2, Rebecca J Richardson1
1School of Physiology, Pharmacology and Neuroscience, Faculty of Biomedical Sciences, University of Bristol, Bristol BS8 1TD, UK.
Disease Models & Mechanisms
|February 27, 2023
Summary
Precise genome editing (PGE) enhances CRISPR technology for modeling human genetic diseases by improving efficiency in creating single-base substitutions. This review details recent advancements in PGE methods for more accurate disease modeling.
Area of Science:
- Molecular Biology
- Genetics
Background:
- CRISPR/Cas gene editing efficiently creates gene disruptions (indels) in model organisms.
- Human genetic diseases often stem from subtle single-base substitutions, requiring precise editing.
- Current precise genome editing (PGE) methods have low efficiency compared to indel generation.
Approach:
- Optimizing guide RNA and donor DNA template design.
- Modulating DNA repair pathways to enhance edit outcomes.
- Developing Cas9 fusion proteins for alternative editing mechanisms.
Key Points:
- Recent progress focuses on improving the efficiency of precise genome editing (PGE).
- PGE advancements are crucial for accurately modeling human genetic diseases.
- Optimizations target guide RNA, DNA templates, repair pathways, and Cas9 fusion proteins.
Conclusions:
- Enhanced PGE methods are vital for creating accurate models of human genetic diseases.
- Continued research into PGE optimization promises significant advancements in disease modeling and research.
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