CRISPR-Based Gene Editing in Human Medicine: Clinical Potentials and Ethical Dilemmas | ||
| Benha Medical Journal | ||
| Volume 42, Issue 10, October 2025, Pages 71-97 PDF (1.29 M) | ||
| Document Type: Review Article | ||
| DOI: 10.21608/bmfj.2025.400323.2518 | ||
| Authors | ||
| Shofia Saghya Infant; Sundaram V* ; Bhavani Sowndharya B; Mathan Muthu C M; Saravanan A; Jenila Rani D | ||
| Department of Biotechnology, Saveetha School of Engineering, Saveetha Institute of Medical and Technical Sciences, Chennai, 602105, India | ||
| Abstract | ||
| CRISPR-mediated gene editing has already revolutionized human medicine with its unprecedented precision and efficiency in editing genetic mutations. Technologically, it is CRISPR-Cas9-based and can introduce precise genome modifications, and has therapeutic potential for monogenic diseases, cancers and infectious diseases. On the clinical front, such results have indicated that certain early-phase trials for β-thalassemia and sickle cell disease using CRISPR-Cas9 have achieved up to 90% decrease in the dependency on transfusions and more than 85% increase in haemoglobin levels, which are tangible results. In the oncologic setting, CRISPR-modified T cells have been employed in immunotherapy trials, and have demonstrated enhanced tumour killing of some hematopoietic malignancies. In addition, infusion of SG cells has allowed editing of hematopoietic stem cells outside the human body, in a controlled and relatively safe setting, a condition absolute for clinical translation. Yet, the technology has significant ethical issues, particularly germline editing, long-term off-target effects, and accessibility. A case in point involves the CRISPR-edited embryos scandal in China, which revealed the absence of a worldwide agreement on regulation as well as the dangers of premature clinical application. Similarly, the cost of CRISPR-based therapies ($1 million plus per patient) serves to widen that divide, especially in LIMCs. As clinical uses extend, there is an urgent need to develop strong ethical frameworks and ways to monitor long-term safety, and to ensure that the technologies are equitably accessible, to incorporate CRISPR into health systems responsibly. | ||
| Keywords | ||
| Health equity; Genetic disorders; β-thalassemia; Sickle cell disease; Immunotherapy | ||
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