Applications and Ethics of Gene Editing Technology in Genetic Disease Treatment

Abstract

Gene editing technology is fundamentally reshaping the paradigm of genetic disease treatment, and its clinical translation marks the entry of modern medicine into an era of precise intervention. The CRISPR-Cas9 system, with its programmable targeting capability, provides radical solutions for monogenic genetic diseases, achieving historic breakthroughs in the treatment of thalassemia and sickle cell disease. Technological iterations have spawned new paradigms of base editing and prime editing, enabling single-base precise correction while circumventing double-strand break risks, representing a qualitative leap in the safety dimension. Ethical controversies focus on the unpredictability of off-target effects, the intergenerational responsibility boundaries of germline editing, and the social equity of technology benefit distribution, urgently requiring the establishment of global collaborative governance mechanisms. Innovations in delivery systems have broken through organ-targeting bottlenecks, making in vivo in situ editing possible. Future development directions lie in integrating artificial intelligence to optimize editing design, perfecting lifelong safety monitoring systems, constructing ethical consensus across multicultural backgrounds, promoting deep synergy between technological innovation and humanistic care, and ultimately achieving a paradigm shift in genetic disease treatment from symptom management to etiological eradication.