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CRISPR/Cas-Based Modifications for Therapeutic Applications: A Review
Nagaraj Bharathkumar1, Abraham Sunil2, Prabhakar Meera1
1B Aatral Biosciences Private Limited, Bangalore, Karnataka, India.
Molecular Biotechnology
|November 6, 2021
Summary
The CRISPR-Cas system, a bacterial immune mechanism, enables precise DNA/RNA editing. This review explores Cas9, Cas12a, and Cas13 variants for therapeutic applications, focusing on future efficacy and safety.
Area of Science:
- Molecular Biology
- Genetics
- Biotechnology
Background:
- CRISPR-Cas is a bacterial immune system targeting foreign DNA/RNA using guide RNA and Cas proteins.
- Its discovery revolutionized genome editing, overcoming limitations of prior tools.
- Cas9, Cas12a, and Cas13 are prominent CRISPR-associated proteins utilized in diverse applications.
Purpose of the Study:
- To review recent advancements in CRISPR-Cas systems for gene modification.
- To focus on Cas proteins like Cas9, Cas12a, and Cas13 for therapeutic potential.
- To explore modifications of Cas proteins for enhanced efficacy and safety in medicine.
Main Methods:
- Systematic review of scientific literature on CRISPR-Cas systems.
- Analysis of Cas protein variants and their applications in research, medicine, and diagnostics.
- Evaluation of modifications aimed at improving therapeutic utility.
Main Results:
- Cas9, Cas12a, and Cas13 are versatile tools for gene editing in various fields.
- Significant progress has been made in developing modified Cas proteins.
- CRISPR technology shows great promise for research applications.
Conclusions:
- CRISPR-Cas systems offer powerful genome editing capabilities.
- The next critical step for CRISPR is demonstrating clinical efficacy and safety.
- Further research into Cas protein variants and modifications is essential for therapeutic translation.
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