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青少年糖尿病研究基金会伴有糖尿病的胰腺器官捐赠者网络(nPOD)计划:目标、运作模式和新发现。

The Juvenile Diabetes Research Foundation Network for Pancreatic Organ Donors with Diabetes (nPOD) Program: goals, operational model and emerging findings.

机构信息

Diabetes Research Institute, University of Miami Miller School of Medicine, Miami, FL, USA; Department of Medicine, Division of Diabetes, Endocrinology and Metabolism, University of Miami Miller School of Medicine, Miami, FL, USA; Department of Immunology and Microbiology, University of Miami Miller School of Medicine, Miami, FL, USA.

出版信息

Pediatr Diabetes. 2014 Feb;15(1):1-9. doi: 10.1111/pedi.12097. Epub 2013 Dec 10.

Abstract

nPOD actively promotes a multidisciplinary and unbiased approach toward a better understanding of T1D and identify novel therapeutic targets, through its focus on the study of human samples. Unique to this effort is the coordination of collaborative efforts and real-time data sharing. Studies supported by nPOD are providing direct evidence that human T1D isa complex and heterogeneous disease, in which a multitude of pathogenic factors may be operational and may contribute to the onset of the disease. Importantly, the concept that beta cell destruction is almost completed and that the autoimmune process is almost extinguished soon after diagnosis is being challenged. nPOD investigators are exploring the hypothesis that beta cell dysfunction may also be a significant cause of hyperglycemia, at least around the time of diagnosis, and are uncovering novel molecules and pathways that are linked to the pathogenesis and etiology of human T1D. The validation of therapeutic targets is also a key component of this effort, with recent and future findings providing new strategic direction for clinical trials.

摘要

nPOD 通过专注于人类样本的研究,积极推动多学科和公正的方法,以更好地理解 T1D 并确定新的治疗靶点。这项工作的独特之处在于协调协作和实时数据共享。nPOD 支持的研究提供了直接证据,证明人类 T1D 是一种复杂和异质的疾病,其中多种致病因素可能起作用,并可能导致疾病的发生。重要的是,β细胞破坏几乎完成,自身免疫过程在诊断后不久几乎消失的概念正受到挑战。nPOD 研究人员正在探索β细胞功能障碍也可能是导致高血糖的一个重要原因的假设,至少在诊断时是这样,并且正在发现与人类 T1D 的发病机制和病因相关的新分子和途径。治疗靶点的验证也是这项工作的一个关键组成部分,最近和未来的发现为临床试验提供了新的战略方向。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/19fa/4282794/dfac0b857983/pedi0015-0001-f1.jpg

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