Epistora Journal of Biomedical Sciences and Research
Current Issue

The discovery and development of CRISPR-Cas9 has revolutionized the field of gene editing, offering unprecedented precision, efficiency, and accessibility for therapeutic applications. This comprehensive review examines the evolution of gene editing technologies from early zinc finger nucleases to the current advanced CRISPR systems. We analyze the fundamental mechanisms of CRISPR-Cas9 and its variants, including base editing, prime editing, and Cas12/Cas13 systems. Key therapeutic applications are discussed across monogenic disorders, cancer immunotherapy, and infectious diseases. Critical challenges addressed include delivery strategies, specificity and off-target effects, immunogenicity, and regulatory considerations for clinical translation. Recent breakthroughs in in vivo delivery, tissue-specific targeting, and multiplexed editing are highlighted as promising developments. The review also presents successful clinical trials and case studies, including ongoing gene therapy initiatives for sickle cell disease, β-thalassemia, and inherited retinal disorders. A strategic framework for accelerating the translation of gene editing technologies from bench to bedside is proposed, emphasizing the importance of improved delivery systems, international collaboration, and ethical regulatory pathways.