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基因编辑胰岛细胞移植通过功能验证
Huan Qiu Wang Zi Xun· 2025-08-08 02:37
Core Viewpoint - The research team from Uppsala University has successfully demonstrated the survival and functionality of donor pancreatic β cells edited with CRISPR-Cas technology in a type 1 diabetes patient without the use of immunosuppressants, marking a significant breakthrough in potential treatments for the disease [1][2]. Group 1: Research Findings - The CRISPR-Cas12b gene editing technique was utilized 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, and the entire procedure was conducted without the use of 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 glycated hemoglobin levels significantly decreased by 42% [2]. - Although the number of transplanted cells was only 7% of the required amount for treatment, this experiment represents a critical breakthrough in the field [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 treatment solution that does not require immunosuppression [2]. - This advancement could relieve millions of patients from the daily burden of insulin injections and the long-term complications associated with type 1 diabetes [2].