CRISPR-Cas9, pharmaceutical applications, chimeric antigen receptor T cell
Abstract
Originally derived from the immune system of bacteria, CRISPR-Cas9 technology has rapidly developed into a revolutionary gene editing tool that is widely used in molecular biology, drug development, and gene therapy. This technology not only provides a new treatment approach for genetic diseases such as sickle cell anemia and Duchenne muscular dystrophy, but also brings breakthrough progress in cancer treatment by enhancing chimeric antigen receptor T cell (CAR-T) therapy. CRISPR-Cas9 has also shown great potential in the drug discovery stage, especially in gene screening, development of disease models, and drug resistance research. However, with the rapid development of CRISPR technology, the scientific community still needs to solve challenges such as potential off-target effects in gene editing, inefficient delivery systems, and long-term safety. In addition, the ethical issues of gene editing have also sparked widespread discussion, especially how to ensure fair access when treating genetic diseases. Despite many challenges, CRISPR-Cas9 technology still shows great application prospects in the future of drug development and gene therapy.
