Comprehensive Analysis of CRISPR-Cas9 Mediated Gene Therapy for Monogenic Disorders

Gene therapy has emerged as a promising approach for the treatment of monogenic disorders, leveraging advances in genome editing technologies. This study aims to evaluate the efficacy and safety of CRISPR-Cas9 mediated gene therapy in correcting genetic mutations responsible for inherited diseases. We employed an in vivo mouse model with a targeted mutation known to cause severe immunodeficiency. The CRISPR-Cas9 system was delivered using an adeno-associated viral vector, and successful gene correction was confirmed through next-generation sequencing. Our results demonstrate a 78% correction rate of the mutated gene and a significant improvement in immune function, as evidenced by a 65% increase in T-cell counts. Importantly, off-target effects were minimal, occurring in less than 0.5% of the genome, suggesting a high degree of specificity. These findings highlight the potential of CRISPR-Cas9 as a viable therapeutic tool for genetic diseases. Further studies are warranted to assess long-term outcomes and refine delivery methods. This research contributes to the growing body of evidence supporting CRISPR-Cas9 as a transformative technology in genetic medicine.