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@article{204776,
        author = {Onkar S. Muppidwar and Vanshita V. kukde and Himanshu B. Tapase and Vaishnavi V. Sur},
        title = {CRISPR-BASED GENE EDITING: A NOVEL PHARMACOLOGICAL APPROACH FOR CYSTIC FIBROSIS TREATMENT},
        journal = {International Journal of Innovative Research in Technology},
        year = {2026},
        volume = {13},
        number = {1},
        pages = {3896-3905},
        issn = {2349-6002},
        url = {https://ijirt.org/article?manuscript=204776},
        abstract = {Cystic fibrosis (CF) remains a serious life-threatening genetic disorder despite advances in CFTR modulator therapies. Approximately 10% of CF patients lack effective pharmacological options due to rare or nonsense mutations. CRISPR-based gene editing technologies offer transformative potential by directly correcting CFTR mutations at the genomic level, providing durable or curative therapeutic alternatives. This comprehensive review synthesizes current knowledge on CRISPR/Cas9, prime editing, and base editing approaches for CF, emphasizing recent preclinical advances in delivery systems, editing eGciency, and functional validation in patient-derived models. Particularly promising are Lung Selective Organ Targeting (SORT) lipid nanoparticles (LNPs) achieving ~60% lung basal cell transfixion and restoring CFTR function in patient-derived bronchial epithelial cells with 16% gene correction eGciency.   Editing modifications, such as 4-FEN (FEN1 nuclease fusion to PEmax), demonstrate enhanced specificity with minimal off-target effects. This review discusses current clinical translation challenges, safety considerations, and the potential for combining CRISPR gene editing with existing CFTR modulators. We also examine emerging strategies including base editing, pegRNA optimization, and non-viral delivery technologies that collectively represent the next frontier in CF therapeutics, potentially enabling the first disease-modifying or curative treatments for all CF genotypes by the early 2030s.},
        keywords = {CRISPR/Cas9, gene editing, cystic fibrosis, CFTR, prime editing, lipid nanoparticles, delivery, gene therapy},
        month = {June},
        }