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Updated: Mar 12, 2026

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Genome Editing in Mammalian Cell Lines using CRISPR-Cas
Published on: April 11, 2019
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Integrating CRISPR/Cas technology with clinical trials: Principles, progress and challenges
Piao Yang1, Mohadeseh Khoshandam2,3, Iman Bhia4
1Department of Molecular Genetics, College of Arts and Sciences, The Ohio State University, Columbus OH 43210, USA.
Asian Journal of Pharmaceutical Sciences
|March 11, 2026
Summary
CRISPR gene editing offers revolutionary potential for treating genetic disorders, neurological diseases, and cancer. This review highlights innovative therapeutic designs and efficiency improvements for CRISPR-based treatments, addressing future challenges in clinical applications.
Area of Science:
- Biotechnology
- Genomics
- Molecular Biology
Background:
- CRISPR gene editing technology has revolutionized genetic modification.
- Its application holds significant promise for treating genetic disorders, neurological diseases, infectious diseases, and cancer.
Purpose of the Study:
- To review current clinical trials utilizing the CRISPR/Cas system for various diseases.
- To emphasize innovative targeting strategies and therapeutic designs for CRISPR applications.
- To explore advancements in CRISPR editing efficiency, challenges, and future potential in managing genetic disorders and cancer.
Main Methods:
- Systematic review of current clinical trials involving CRISPR/Cas technology.
- Analysis of recent research focusing on CRISPR editing efficiency and therapeutic design.
- Examination of innovative targeting conditions and approaches for CRISPR-based therapies.
Main Results:
- CRISPR technology is rapidly expanding, with ongoing clinical trials exploring its therapeutic potential.
- Emphasis is placed on novel targeting strategies and functional therapeutic designs beyond traditional delivery methods.
- Research is actively increasing CRISPR editing efficiency for genetic disorders and cancer management.
Conclusions:
- CRISPR-based therapies show transformative potential for future medical treatments.
- Continued research into efficiency, targeting, and overcoming challenges is crucial for expanding clinical applications.
- This review provides a unique perspective on advancing CRISPR technology for improved patient outcomes.
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