Sana Biotechnology(US:SANA) 量子位·2025-09-16 05:58Core Viewpoint - The article highlights a significant breakthrough in diabetes treatment, where CRISPR-edited pancreatic cells were successfully transplanted into a type 1 diabetes patient, showing promising results in insulin secretion and immune evasion [1][2][3]. Group 1: Research Background - Type 1 diabetes is an autoimmune disease where the immune system attacks insulin-secreting pancreatic cells, leading to uncontrolled blood sugar levels [4][5]. - The research conducted by Sana Biotechnology aims to provide a potential cure for approximately 9.5 million type 1 diabetes patients globally [8]. Group 2: Methodology - Researchers extracted pancreatic cells from a 60-year-old deceased donor and utilized CRISPR-Cas12b technology to edit these cells by knocking out two key genes, B2M and CIITA, which typically mark foreign invaders for the immune system [9][10]. - To further protect the cells from immune surveillance, a gene encoding the CD47 protein was introduced, which sends a "don't eat me" signal to the immune system [12]. Group 3: Clinical Application - The edited pancreatic cells, totaling 79.6 million, were implanted into a 42-year-old patient with 37 years of type 1 diabetes through 17 injections into muscle tissue [20][24]. - Notably, the entire procedure did not involve any glucocorticoids, anti-inflammatory drugs, or immunosuppressants [25]. Group 4: Results and Future Plans - After 12 weeks post-transplant, the edited cells showed no signs of rejection and continued to secrete insulin, effectively regulating the patient's blood sugar levels [26]. - C-peptide levels, a direct marker of endogenous insulin secretion, were significantly elevated at 4, 8, and 12 weeks post-intervention [28]. - Sana Biotechnology plans to conduct more comprehensive clinical trials starting next year to further investigate the treatment's efficacy [30].