首次人体实验成功，基因编辑胰岛细胞“隐身”植入，可正常分泌胰岛素

Core Insights - A breakthrough in diabetes treatment has been achieved with the first successful transplantation of CRISPR-edited pancreatic cells into a type 1 diabetes patient, allowing for insulin secretion without the need for immunosuppressants [1][2]. Group 1: Research and Methodology - The study published in the New England Journal of Medicine and reported on Nature's website highlights the potential of CRISPR gene editing in treating type 1 diabetes, which affects approximately 9.5 million patients globally [2]. - 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 signal T cells to attack foreign invaders [2][3]. - To further protect the edited cells from immune system attacks, a gene encoding the CD47 protein was introduced, which sends a "do not eat me" signal to the immune system [2]. Group 2: Clinical Application - The final cell preparation, named UP421, consisted of three types of cells: fully edited cells lacking HLA and expressing high levels of CD47, partially edited cells with some HLA and maintaining endogenous CD47 levels, and wild-type cells with varying CD47 levels [6]. - The edited pancreatic cells were implanted into a 42-year-old patient with 37 years of type 1 diabetes through 17 injections, totaling 79.6 million engineered cells [8][11]. - Remarkably, the entire procedure did not involve any glucocorticoids, anti-inflammatory drugs, or immunosuppressants, and after 12 weeks, the cells showed no signs of rejection while effectively regulating the patient's blood sugar levels [12]. Group 3: Results and Future Plans - C-peptide levels, a direct marker of endogenous insulin secretion, were undetectable at baseline but showed significant increases at weeks 4, 8, and 12 post-intervention, indicating successful insulin production [13]. - Even six months post-transplant, the edited cells continued to evade immune detection and attack [13]. - However, the study involved only one participant, and the treatment duration was insufficient to eliminate the need for insulin injections, prompting the company to plan further clinical trials starting next year for more comprehensive research [14].