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CRISPR-Cas9 in genome editing: Its function and medical applications
Saedeh Khadempar1, Shokoufeh Familghadakchi2, Roozbeh Akbari Motlagh3
1Departemant of Medical Genetics, Shahid Sadoughi University of Medical Science, Yazd, Iran.
Journal of Cellular Physiology
|October 27, 2018
Summary
Clustered regularly interspaced short palindromic repeats (CRISPR)-Cas9 technology offers a precise and efficient method for genome editing. Ongoing research enhances its accuracy and expands its therapeutic applications for genetic disorders.
Area of Science:
- Molecular Biology
- Genetics
- Biotechnology
Background:
- RNA-guided nucleases enable rapid, efficient genome modification for gene studies and disease research.
- CRISPR-Cas9, a bacterial immune system, functions as molecular scissors for targeted genome editing.
- Existing technologies include zinc finger nucleases, TALENs, and CRISPR-Cas9.
Purpose of the Study:
- To review the history and direct aspects of CRISPR-Cas9 technology.
- To highlight its precision in genomic targeting and control over correction events.
- To discuss its applications in biological studies and disease treatment.
Main Methods:
- Review of CRISPR-Cas9 system history and advancements.
- Analysis of guide RNA selection and enzyme engineering.
- Examination of methods for identifying off-target effects.
Main Results:
- CRISPR-Cas9 offers precise genome targeting for gene manipulation.
- Advances have been made in enhancing Cas9 specificity and reducing off-target mutations.
- The technology shows potential for correcting disease-causing mutations.
Conclusions:
- CRISPR-Cas9 is a powerful tool for genetic research and therapeutic development.
- Continued improvements in precision and delivery are crucial for clinical applications.
- This technology holds significant promise for future biological studies and modern disease treatment.
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