基因编辑胰岛细胞移植通过功能验证 为Ⅰ型糖尿病治疗开辟新途径

Core Viewpoint - The research team from Uppsala University has successfully demonstrated the world's first instance of donor pancreatic β cells edited by CRISPR-Cas technology surviving and functioning for 12 weeks in a type 1 diabetes patient without the use of immunosuppressants, offering hope for a potential cure for the disease [1][2]. Group 1: Research Breakthrough - The study utilized CRISPR-Cas12b technology to modify donor pancreatic cells by knocking out B2M and CIITA genes to reduce immunogenicity and overexpressing CD47 through lentiviral transduction [1]. - The modified low-immunogenicity cells were implanted into a 42-year-old subject's left brachioradialis muscle, with the entire procedure conducted without glucocorticoids, anti-inflammatory drugs, or immunosuppressants [1]. Group 2: Clinical Outcomes - Monitoring over 84 days showed that the transplanted cells successfully evaded the immune system, with no T cell activation or antibody response observed [2]. - Imaging confirmed the good survival of the graft, and the patient's hemoglobin A1c levels significantly decreased by 42% [2]. - Although the number of transplanted cells was only 7% of what is needed for treatment, this experiment marks a critical breakthrough in demonstrating survival and functionality [2]. Group 3: Future Implications - The low-immunogenic gene editing technology used in this study may lead to the development of more curative β cells, potentially allowing for a diabetes cure without the need for immunosuppression [2]. - This advancement could relieve millions of patients from the daily pain of injections and the long-term complications associated with diabetes [2].