CRISPR-Cas9 PROTEIN: PHARMACEUTICAL FRONTIERS IN GENETIC RESTORATION

The advent of CRISPR-Cas9 has revolutionized gene editing, offering vast possibilities for disease treatment. This review dissects its mechanisms, structural aspects, and classification, underlining its superiority to preceding technologies such as ZFNs and TALENs. The evolution of CRISPR’s applications is traced from bacterial immunity to the forefront of genome editing. Delivery methods—both viral and non-viral—are assessed, with detailed exploration of emerging solutions like lipid nanoparticles and DNA nanocarriers. CRISPR/Cas9 demonstrates significant therapeutic value in oncology by inactivating oncogenes, restoring tumor suppressors, and enhancing immunotherapeutic strategies. Furthermore, promising results have emerged in treating inherited disorders such as beta-thalassemia and sickle cell anaemia, as well as in clinical studies focused on T-cell therapies and viral diseases. Although these developments are encouraging, obstacles persist, including off-target genetic modifications and the intricacies of delivery. Innovations, notably base editing and prime editing, show promise to resolve these barriers. This review highlights the immense therapeutic prospects of CRISPR/Cas9 and stresses the importance of sustained research to fully unlock its clinical benefits.