CRISPR -based gene therapy and genome editing in drug design

Abstract

Genome editing has transformed molecular biology by enabling precise modification of genetic material and advancing drug development. The evolution from homologous recombination, Zinc Finger Nucleases (ZFNs), and Transcription Activator-Like Effector Nucleases (TALENs) to CRISPR-Cas systems, particularly CRISPR-Cas9, has significantly improved genome editing due to its simplicity, efficiency, and versatility. Advanced technologies such as CRISPR-Cas12a, base editing, prime editing, and RNA-targeting Cas13 have further enhanced editing precision while expanding applications beyond DNA modification. Genome editing has shown great potential in treating hereditary diseases, infectious diseases, and cancer, supporting personalized medicine and therapeutic innovation. Efficient viral and non-viral delivery systems remain essential for successful clinical application. Despite challenges such as off-target effects, immunogenicity, germline ethics, affordability, and equitable access, CRISPR-based technologies continue to redefine biomedical research and next-generation genetic medicine. Responsible collaboration among researchers, policymakers, and society is essential for safe and ethical implementation.