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CRISPR/Cas9 technology as a potent molecular tool for gene therapy
Ansar Karimian1,2,3, Khalil Azizian4, Hadi Parsian1
1Cellular and Molecular Biology Research Center, Health Research Institute, Babol University of Medical Sciences, Babol, Iran.
Journal of Cellular Physiology
|January 31, 2019
Summary
Clustered regularly interspaced short palindromic repeats/CRISPR-associated nuclease 9 (CRISPR-Cas9) gene editing offers a cost-effective and flexible approach for correcting mutations. This review explores its therapeutic potential, particularly in cancer treatment, despite delivery and safety challenges.
Area of Science:
- Molecular Biology
- Biotechnology
- Genetics
Background:
- CRISPR-Cas9 is an RNA-guided gene editing tool.
- It presents advantages over older methods like zinc finger nucleases and transcription activator-like effector nucleases.
- CRISPR-Cas9 is recognized for its cost-effectiveness, flexibility, and ease of use.
Purpose of the Study:
- To review CRISPR-Cas-based gene editing, including its classifications and mechanism of action.
- To discuss the therapeutic applications of CRISPR-Cas9 in treating mutational disorders.
- To emphasize the potential of CRISPR-Cas9 in cancer treatment, alongside its advantages and limitations.
Main Methods:
- This review synthesizes information on CRISPR-Cas9 technology.
- It examines the mechanism of action and classifications of CRISPR-Cas-based gene editing.
- The review analyzes delivery systems and therapeutic applications, focusing on cancer.
Main Results:
- CRISPR-Cas9 is a powerful gene editing tool with significant therapeutic potential.
- It offers advantages in cost, flexibility, and usability compared to conventional methods.
- Challenges related to efficient delivery and safety require further investigation.
Conclusions:
- CRISPR-Cas9 holds promise for treating genetic disorders and cancer by correcting mutations.
- Continued research into delivery systems and safety is crucial for clinical translation.
- CRISPR-Cas9 represents a convenient and powerful tool for gene editing applications.
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