Journal of Basic Research in Medical Sciences
J Basic Res Med Sci
Volume 13, Issue 1 (1-2026)
jbrms 2026, 13(1): 67-84 |
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Alivaisi E, Noori-Zadeh A. Genetic Surgery: The Revolutionary CRISPR-Cas9 Scissors. jbrms 2026; 13 (1) :67-84
URL: http://jbrms.medilam.ac.ir/article-1-1027-en.html
URL: http://jbrms.medilam.ac.ir/article-1-1027-en.html
Department of Clinical Biochemistry, Faculty of Medicine, Ilam University Medical Sciences, Ilam, Iran , alincbc@gmail.com
Abstract: (96 Views)
Introduction: The CRISPR-Cas9 system, a revolutionary genome-editing tool which stems from a bacterial adaptive immune system, shows a paradigm shift in designing new therapeutics in medicine.
Aim: This review describes the CRISPR-Cas9 technology from its initial discovery in prokaryotes as a bacterial adaptive immune system to its development into a precise molecular scalpel capable of human genome editing. It explores CRISPR-Cas9 fundamental components and mechanisms, highlighting how a synergy between a guide RNA and Cas9 nuclease allows targeted DNA double-strand breaks, and harness DNA repair enzymes of the target cell for genetic code modifications.
Discussion: There is a lot of promise for using CRISPR-Cas9 technology to treat diseases. In clinical settings, it has led to breakthroughs in the treatment of monogenic disorders like sickle cell anaemia and beta-thalassemia, and it is also making good progress in oncology and other areas. The technology still has a lot of problems to solve, even with these successes. For example, it can cause mutations that aren't intended, it has technical limits, and there are moral concerns about how it could be misused, such as for heritable human enhancement.
Conclusion: CRISPR-Cas9 technology offers remarkable therapeutic potential with clinical applications already yielding breakthroughs in curing monogenic disorders such as sickle cell anemia and beta-thalassemia, and promising advances in oncology and beyond. However, it also presents significant ethical and practical risks, including the threat of heritable human enhancement (designer babies), and persistent safety concerns such as off-target mutations.
Aim: This review describes the CRISPR-Cas9 technology from its initial discovery in prokaryotes as a bacterial adaptive immune system to its development into a precise molecular scalpel capable of human genome editing. It explores CRISPR-Cas9 fundamental components and mechanisms, highlighting how a synergy between a guide RNA and Cas9 nuclease allows targeted DNA double-strand breaks, and harness DNA repair enzymes of the target cell for genetic code modifications.
Discussion: There is a lot of promise for using CRISPR-Cas9 technology to treat diseases. In clinical settings, it has led to breakthroughs in the treatment of monogenic disorders like sickle cell anaemia and beta-thalassemia, and it is also making good progress in oncology and other areas. The technology still has a lot of problems to solve, even with these successes. For example, it can cause mutations that aren't intended, it has technical limits, and there are moral concerns about how it could be misused, such as for heritable human enhancement.
Conclusion: CRISPR-Cas9 technology offers remarkable therapeutic potential with clinical applications already yielding breakthroughs in curing monogenic disorders such as sickle cell anemia and beta-thalassemia, and promising advances in oncology and beyond. However, it also presents significant ethical and practical risks, including the threat of heritable human enhancement (designer babies), and persistent safety concerns such as off-target mutations.
Type of Study: Narrative review |
Subject:
Biochemistry
Received: 2025/09/7 | Accepted: 2025/10/20 | Published: 2026/01/4
Received: 2025/09/7 | Accepted: 2025/10/20 | Published: 2026/01/4
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