CRISPR-Based Gene Editing in Disease Treatment: Ethical and Practical Challenges

Abstract

CRISPR-based gene editing has rapidly emerged as a transformative technology in modern medicine, offering unprecedented precision in correcting genetic mutations and treating complex diseases. This study employed a mixed-methods framework integrating experimental analysis, computational modeling, and ethical inquiry to evaluate both the therapeutic potential and the challenges of CRISPR applications in disease treatment. Experimental results demonstrated marked improvements in editing efficiency and reductions in off-target events through optimized delivery systems and engineered Cas variants, while computational modeling provided predictive insights into repair pathway dynamics and mutation probabilities. However, persistent variability across trials highlights the continued technical limitations that must be addressed before widespread clinical translation is possible. Complementing these findings, a qualitative review of ethical and policy frameworks revealed broad support for somatic editing but strong global resistance to germline modification due to intergenerational risks, consent concerns, and potential exacerbation of social inequities. The study further identified distributive justice and equitable access as central challenges, with evidence suggesting that CRISPR therapies risk widening global health disparities without deliberate governance measures. Taken together, the results underscore that CRISPR’s clinical promise is inseparable from its ethical and regulatory context. Achieving safe and equitable implementation will require not only scientific innovation but also anticipatory governance, international collaboration, and inclusive stakeholder engagement. This integrated approach will be essential to ensure that CRISPR becomes a tool for collective health benefit rather than a source of new medical and social divisions.