CRISPR-Cas9 in Pharmaceutical Research: Applications, Challenges, Ethical Considerations and Future Directions

The precise genome editing capabilities of CRISPR-Cas9 have transformed pharmaceutical research. This potent tool could influence drug development, disease modelling, and gene therapy. CRISPR-Cas9, originating from the immune system of bacteria, has transformed genetic engineering through enhanced precision in gene modification. Advancements have enhanced the comprehension of genetic disorders, the treatment of complex diseases, and the improvement of diagnostics. CRISPR is utilised in drug development through functional genomics and disease models to identify targets, accelerate drug discovery, and enhance the understanding of disease mechanisms. CRISPR therapies demonstrate the system's capacity to address incurable diseases such as sickle cell disease, beta-thalassemia, and specific cancers. CRISPR in clinical applications encounters obstacles, such as off-target effects, limitations in delivery mechanisms, and the necessity for scalable production for extensive clinical utilisation. Ethical considerations include the risks of human germline editing, equitable access to CRISPR-based therapies, and obtaining informed consent from participants in experimental treatments. This review elucidates the advantages, disadvantages, and ethical considerations of CRISPR. It proposes research avenues and underscores the necessity for a definitive regulatory framework and global ethical standards for the application of CRISPR in pharmaceutical research.