Advancements in CRISPR/Cas Technology for Gene Therapy
Author Information
Author(s): Bairqdar Ahmad, Karitskaya Polina E., Stepanov Grigory A.
Primary Institution: Institute of Chemical Biology and Fundamental Medicine of the Siberian Branch of the Russian Academy of Sciences
Hypothesis
This review examines the current landscape of CRISPR–Cas implementation in clinical trials for treating genetic disorders, cancers, and infectious diseases.
Conclusion
CRISPR/Cas technology has marked a pivotal shift in genetic medicine, enabling precise, targeted treatments for previously intractable genetic disorders.
Supporting Evidence
- CRISPR–Cas technology has transformed the field of gene editing, opening new possibilities for treatment of various genetic disorders.
- Recent years have seen a surge in clinical trials using CRISPR–Cas-based therapies.
- This review emphasizes the mechanism of action of tested therapies and the delivery method.
Takeaway
CRISPR is like a pair of molecular scissors that can cut DNA to fix genetic problems, helping treat diseases that were hard to manage before.
Methodology
The review analyzes data from clinical trials and key registries to explore the application of CRISPR–Cas systems in various therapeutic contexts.
Limitations
The long-term effects of CRISPR/Cas are still poorly understood, and there are concerns about off-target effects and mosaicism.
Digital Object Identifier (DOI)
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