The Role and Impact of CRISPR-Cas9 Gene Editing in the Treatment of Genetic Diseases: Challenges and Perspectives

Introduction: CRISPR-Cas9 gene editing technology has emerged as one of the most advanced biotechnological tools with significant potential for treating genetic diseases. This review examines the efficacy, challenges, and future prospects of this technology in the treatment of genetic disorders. Methods: Relevant articles on CRISPR-Cas9 were retrieved from reputable databases, including PubMed, Springer, and Nature. Selection criteria included publication in high-impact journals between 2014 and 2024 and a focus on the application of this technology in genetic diseases. The collected data were analyzed and compared based on mechanisms of action, laboratory and clinical outcomes, and existing challenges. Results: CRISPR-Cas9 has demonstrated its ability to correct defective genes in disorders such as Duchenne muscular dystrophy, sickle cell anemia, and beta-thalassemia. Studies indicate that this technology has been successful in restoring muscle function and increasing healthy hemoglobin levels. Additionally, in hereditary cancers such as lung and breast cancer, CRISPR has been used to suppress tumor-promoting genes, leading to tumor growth reduction in animal models. However, several challenges remain, including off-target effects, delivery system limitations, and ethical and regulatory concerns, which restrict its widespread clinical application. Conclusion: As a revolutionary genome-editing tool, CRISPR-Cas9 holds the potential to fundamentally transform the treatment of genetic diseases. Future advancements in enhancing precision, minimizing side effects, and addressing ethical dilemmas could pave the way for its broader clinical adoption. This technology offers promising prospects for personalized medicine and genome-based therapies.