Current drug targets2026ResearchNon-viral Gene Delivery
1. CRISPR/Cas9-based Genome Editing in Inherited Diseases: Mechanisms, Therapeutic Advances, Safety and Clinical Translation
Fatima J, Siddique Yh
Gene therapy has transformed modern biomedical research by enabling curative strategies that target the molecular basis of disease. Among genome-editing technologies, the CRISPR/Cas9 system has emerged as a versatile and efficient platform due to its programmability, simplicity, and precision. This review summarizes…
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