TCR-T细胞,1型糖尿病 z-bo 2020-10-07 340

　　Dr. Davidollings, director of the Immunotherapy Center at Seattle Children's Research Institute, dreams of developing a treatment for children with type 1 diabetes for most of the past decade. The therapy does not require insulin injections and uses humans. Unique immune cells targeted for treatment of this disease. Now, with new research and new funding, this dream is about to come true, and the first clinical trials of human experimental treatments have begun. The clinical trial will be conducted by the Seattle Children's Research Institute in collaboration with Benaroya Research Institute.

　　Awlings said: "The original dream can now be realized. We hope that our research will lead to new therapies that can prevent the destructive immune response that leads to type 1 diabetes in children."

　　is type 1. In diabetes, certain types of immune cells (called effector T cells) mistakenly attack the islet cells of the pancreas that produce insulin. The job of these islet cells is to sense elevated blood sugar levels and respond by releasing insulin. Because other components of the immune system (regulatory T cells (Tregs)) are not functioning properly, this attack continues. When destroyed by uncontrolled effector T cells, these islet cells cannot release insulin. Blood sugar levels continue to rise, leading to early symptoms of diabetes such as frequent urination, thirst, hunger, and extreme fatigue.

　　New genetically modified T cells provide a way to restore pancreas balance

　　To prevent this attack, Rollings Institute is a method to genetically modify the patient's own T cells to make them look normal. Treg cells can also work normally. When they are transplanted back into the patient, these genetically modified or edited regulatory T cells (edTregs) enter the pancreas, where they can suppress the overactive immune response to maintain and protect these islets. I want cell function.

　　This article shows how these researchers used gene editing technology to target the FOXP3 gene in human T cells. By activating FOXP3, we provide T cells with the instructions needed to form Treg cells.

　　The edTreg cells produced by

　　are very similar to natural Treg cells. When tested in animal models and tissue cultures, its function is also similar to that of natural Treg cells. Finally, these researchers showed how to make these transgenic cells antigen-specific. According to Rawlings, this function is achieved by attaching T cell receptors (TCR) to the surface of these genetically modified cells. This is important for introducing these cells into the pancreas of diabetic patients. .. Research to further validate these results will help pave the way for phase I clinical trials of cell therapy for type 1 diabetes. "These data confirm for the first time that genetic modification by activating FOXP3 is sufficient to generate functional edTreg cells. This is just a groundbreaking research finding. On the contrary, it can also be directly translated into clinical use.

　　have a bright future

　　In the next phase, these researchers will fine-tune the T cell receptors needed to make edTreg. Efforts to improve the production process of edTreg cells for clinical use.

　　Rollins said: "In collaboration with researchers from the Benaroya Institute, we have combined extensive expertise in the discovery and testing of T cell receptors in diabetic patients with new technologies for T cell genetic modification."

　　Best T cell receptor guides edTreg cells to the pancreas and activates its protective activity. Ideally, this also benefits most type 1 diabetes patients. We want to identify T cell receptors to construct recombinant Treg cells to protect the pancreas. This treatment can prevent the destruction of insulin-producing cells, thereby slowing the progression of the disease. , Which can ultimately be used for prevention.