Core Viewpoint - The recent successful application of personalized gene editing therapy on a 6-month-old infant marks a significant milestone in the treatment of genetic diseases, opening new avenues for patients lacking effective treatment options [1] Group 1: Gene Editing Technology Overview - Gene editing technology allows for precise deletion, insertion, or replacement of specific genes, akin to a "molecular scissors" that can correct and modify defective genes [2][4] - Unlike transgenic technology, which randomly integrates foreign genes into an organism's genome, gene editing modifies the organism's own genes without disrupting the overall structure [2][4] - The evolution of gene editing technology has progressed rapidly, particularly since the advent of CRISPR technology in 2012, which has simplified the process and significantly reduced costs [5][6] Group 2: Applications in Medicine - Gene editing technology is being applied in the treatment of genetic diseases, such as thalassemia, where CRISPR can edit a patient's hematopoietic stem cells to restore normal gene expression [7] - In cancer treatment, CAR-T therapy utilizes gene editing to enhance the immune cells' ability to combat cancer cells, demonstrating the technology's potential in oncology [7] - The technology also aids in modeling complex diseases in research, accelerating drug development by allowing scientists to observe disease progression in genetically edited organisms [7] Group 3: Applications in Agriculture and Bio-manufacturing - In agriculture, gene editing has led to the development of new rice varieties that are resistant to diseases and environmental stress, contributing to global food security [8] - In bio-manufacturing, gene editing enhances production efficiency and reduces costs, such as in the production of biofuels and scarce pharmaceuticals [8] Group 4: Ethical Considerations - The advancement of gene editing technology raises ethical concerns, particularly regarding the editing of human germline cells, which could permanently alter the human gene pool [10] - Ethical guidelines emphasize the importance of prioritizing non-heritable somatic cell editing for therapeutic purposes and prohibiting germline editing in clinical applications [10][11] - The establishment of strict technical boundaries and international regulatory frameworks is essential to prevent ethical violations and ensure that gene editing serves societal welfare [10][11]