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Gene surgery: Potential applications for human diseases
Ayman El-Kenawy1,2, Bachir Benarba3, Adriana Freitas Neves4
1Department of Pathology, College of Medicine, Taif University, Saudi Arabia.
EXCLI Journal
|November 26, 2019
Summary
Gene surgery, particularly using CRISPR-Cas, offers promising therapeutic applications for diseases like cancer and diabetes. However, challenges in design, delivery, and ethical considerations remain for this emerging gene editing technology.
Area of Science:
- Biotechnology
- Genetics
- Medical Science
Background:
- Gene therapy has emerged as a significant therapeutic approach for various diseases.
- CRISPR-Cas technology has advanced gene editing capabilities for eukaryotic cells.
Purpose of the Study:
- To review the applications of gene surgery in treating human diseases.
- To discuss the molecular mechanisms underlying gene surgery's therapeutic effects.
- To highlight the role of CRISPR-Cas in gene surgery.
Main Methods:
- Review of existing studies on gene surgery applications.
- Analysis of molecular mechanisms in gene therapy.
- Focus on CRISPR-Cas, ZFNs, and TALENs technologies.
Main Results:
- Gene surgery shows therapeutic potential in cancer, diabetes (Type 1), cardiovascular diseases, neurological disorders, and β-thalassemia.
- CRISPR-Cas is effective in the diagnosis, screening, and treatment of various cancers.
- Gene surgery effectively targets signaling pathways in Type 1 diabetes.
Conclusions:
- Gene surgery, especially with CRISPR-Cas, presents significant therapeutic potential across multiple human diseases.
- Limitations include design complexities, cost, off-target effects, delivery challenges, and ethical concerns.
- Further research is needed to overcome these limitations for widespread clinical application.
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