Genome Editing with CRISPR-Cas۹ in the Treatment of Hereditary Diseases: Achievements, Technical Challenges, and Ethical Considerations

The advent of CRISPR-Cas۹ technology has revolutionized the field of genetic engineering, offering unprecedented precision in modifying the human genome. By harnessing a bacterial immune defense mechanism, CRISPR-Cas۹ enables targeted double-strand breaks in DNA, facilitating the correction of pathogenic mutations responsible for a wide array of hereditary diseases. This review provides a comprehensive analysis of the current landscape of CRISPR-based therapeutic interventions, highlighting recent clinical successes in treating disorders such as sickle cell disease, B-thalassemia, and Leber congenital amaurosis. Furthermore, the article addresses critical technical challenges that currently hinder widespread clinical application, most notably the risks of off-target effects, mosaicism, and the complexities of efficient in vivo delivery systems. Finally, the paper explores the profound ethical, legal, and social implications associated with genome editing, specifically distinguishing between somatic and germline modifications. By synthesizing technical advancements with ethical frameworks, this review outlines the trajectory of CRISPR-Cas۹ as a transformative tool in precision medicine and underscores the necessity for rigorous safety protocols and global regulatory consensus before its full integration into routine clinical practice.

کلیدواژه ها:

Genome Editing ، Hereditary Diseases ، Precision Medicine ، Gene Therapy ، Off-Target Effects ، Bioethics