CRISPR-Cas9 gene editing in biomedical science

Recognized as a versatile gene-editing platform, the CRISPR-Cas9 system has redefined approaches in biomedical science and the development of novel medical treatments. Through its ability to introduce precise and efficient alterations in genetic material, the system offers significant potential for treating a variety of genetic abnormalities, malignancies, viral infections including HIV, and metabolic and neurodegenerative c onditions. Its simplicity, cost effectiveness, and precision have made it a preferred tool compared with earlier targeted DNA-manipulation techniques, including zinc finger nucleases (ZFNs) and transcription activator-like effector nucleases (TALENs). However, this system also has limitations; challenges such as unintended modifications at non-targeted sites, distribution inefficiencies, activation of the immune system, and unresolved ethical dilemmas, especially regarding germline editing, continue to raise concerns. Additionally, regulatory approaches to CRISPR Cas9 differ across countries, complicating its global implementation. Ongoing research focuses on enhancing the specificity, safety, and delivery of CRISPR based therapies through innovations such as improved guide RNA design, alternative Cas enzymes, and advanced delivery systems. The current review discusses the molecular functioning of CRISPR Cas9 system, its wide-ranging biomedical applications, safety and ethical considerations, and the future directions of this transformative technology in personalized medicine and gene therapy. Overall, these advancements reflect the growing global research and understanding of CRISPR-Cas9 as an accurate platform for DNA manipulation, while also highlighting its transformative biomedical potential and the need to increase awareness and familiarity with such advanced technologies within the Sri Lankan scientific and clinical context.