Gene Editing Techniques in the Treatment of Genetic Disorders

Advancements, Challenges, and Future Directions in Gene Editing for Genetic Disorders

Abstract

This research article explores the application of gene editing techniques, particularly CRISPR-Cas9, in the treatment of genetic disorders. We evaluated the efficacy and safety of various gene editing strategies in preclinical and clinical settings, focusing on their ability to correct genetic mutations responsible for disorders such as cystic fibrosis, muscular dystrophy, and sickle cell anemia. Our findings indicate that CRISPR-Cas9 and other gene editing tools can effectively target and modify defective genes, leading to significant improvements in cellular function and disease symptoms. However, we also address challenges related to off-target effects, delivery mechanisms, and ethical considerations. This study underscores the transformative potential of gene editing technologies while highlighting the need for ongoing research to refine these methods and ensure their safe application in clinical practice.