Published Paper Details:

CRISPR-Cas9 (Clustered Regularly Interspaced Short Palindromic Repeats) represents a transformative advance in molecular biology, reshaping pharmaceutical research through precise and efficient genome editing. This technology enables targeted modification of genetic material in living cells, offering unprecedented potential for the treatment of genetic and complex diseases. CRISPR-Cas9 operates via three core steps: guide RNA-mediated target DNA recognition, Cas9-induced double-strand break formation, and cellular repair through non-homologous end joining or homology-directed repair. Recent innovations, including base editing and prime editing, have significantly improved editing accuracy while reducing unintended genomic alterations. Clinical translation has progressed rapidly, highlighted by the FDA approval of CASGEVY(TM) in December 2023 for sickle cell disease and transfusion-dependent ?-thalassemia--the first approved CRISPR-based therapy. Therapeutic applications now span monogenic disorders, cancer immunotherapy via CAR-T cell engineering, viral infections, and neurological diseases, with ongoing clinical trials targeting HIV, hemophilia, cystic fibrosis, and inherited retinal disorders. Effective delivery remains a major challenge, though adeno-associated viruses, lipid nanoparticles, and lentiviral vectors show clinical promise. Persistent concerns include off-target effects, immunogenicity, scalability, and long-term safety. High-fidelity Cas9 variants and optimized guide RNA chemistries have substantially mitigated these risks. Future directions emphasize multiplexed and epigenetic editing, integration with conventional therapies, and AI-assisted CRISPR design. Collectively, CRISPR-based therapeutics are poised to shift medicine from symptomatic treatment toward durable genetic correction and precision healthcare.