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@article{181821,
        author = {Dr.arfa khan and kailash kumar and jatin upadhyay and kali kumari and Dr.raghvendra singh bhadauria},
        title = {CRISPR-Cas9 Gene Therapy: Revolutionizing the Treatment of Genetic Disorders},
        journal = {International Journal of Innovative Research in Technology},
        year = {2025},
        volume = {12},
        number = {1},
        pages = {5589-5595},
        issn = {2349-6002},
        url = {https://ijirt.org/article?manuscript=181821},
        abstract = {CRISPR-Cas9 has emerged as a groundbreaking genome-editing technology that enables targeted, efficient, and cost-effective modification of genetic material. Originating from a natural bacterial defense system, CRISPR-Cas9 has significantly advanced beyond previous tools such as Zinc Finger Nucleases (ZFNs) and Transcription Activator-Like Effector Nucleases (TALENs), owing to its simplicity, precision, and scalability. The system uses a guide RNA (gRNA) to direct the Cas9 enzyme to specific DNA sequences, where it induces double-strand breaks. These breaks are repaired by cellular mechanisms—non-homologous end joining (NHEJ) or homology-directed repair (HDR) allowing for gene disruption, correction, or insertion. This review explores the mechanism of CRISPR-Cas9 and highlights its therapeutic applications in various genetic disorders, including sickle cell anemia, cystic fibrosis, Huntington’s disease, and Leber congenital amaurosis. In several of these conditions, CRISPR-based therapies have shown promising results in clinical and preclinical studies, leading to breakthroughs such as the FDA-approved Casgevy for sickle cell disease. Emerging technologies like base and prime editing enhance precision by enabling nucleotide-level changes without inducing double-strand breaks. Despite its potential, CRISPR faces significant challenges including off-target effects, delivery limitations, and immune responses. Ethical concerns, particularly regarding germline editing and equitable access, also demand careful regulation and societal dialogue.},
        keywords = {—CRISPR-Cas9, gene editing, genetic disorders, base editing, prime editing, therapeutic applications, off-target effects, ethical considerations},
        month = {June},
        }