CRISPR/Cas9 for Gene Therapy in Inherited Disorders: A Comprehensive Review
Abstract
The CRISPR/Cas9 (Clustered Regularly Interspaced Short Palindromic Repeats-associated protein 9) system has emerged as a revolutionary genome-editing tool, offering unprecedented precision, efficiency, and affordability in genetic engineering. Its application in gene therapy for inherited disorders presents a transformative approach to treating monogenic diseases by directly correcting pathogenic mutations at the DNA level. This review provides an in-depth analysis of the CRISPR/Cas9 mechanism, its therapeutic applications in various inherited disorders, and the latest advancements in delivery systems. We discuss preclinical and clinical successes in treating conditions such as sickle cell anemia, cystic fibrosis, and Duchenne muscular dystrophy, while also addressing key challenges, including off-target effects, immune responses, and ethical concerns. Despite these hurdles, CRISPR/Cas9-based gene therapy holds immense potential for curing previously untreatable genetic diseases. Future research should focus on improving editing precision, optimizing delivery methods, and establishing robust regulatory frameworks to facilitate clinical translation.
How to Cite This Article
Dr. Mohit Bansal (2025). CRISPR/Cas9 for Gene Therapy in Inherited Disorders: A Comprehensive Review . International Journal of Pharma Insight Studies (IJPIS), 2(2), 04-06.