CureGN

摘要 治疗肾小球肾病(CureGN)是一家国家糖尿病、消化和肾脏研究所 疾病(NIDDK)由多中心国际联盟赞助，成立于2013年，旨在研究自然 肾小球病史(微小病变、局灶节段性肾小球硬化、膜性 肾病和IgA肾病)，了解它们的潜在生物学，开发生物标记物来解决 疾病的异质性，并最终改善治疗选择。CureGN在以下方面取得了进展：1) 建立一个庞大的、不同种族的肾小球疾病患者纵向观察队列；2) 创建丰富的、精心策划的临床数据集和相关的生物样本；3)建立最大的 数字肾脏活检；以及4)基础设施，将促进核心和临床研究 辅助研究科学家。 定义CureGN研究的疾病的遗传和基因组图景一直是从 这项研究的启动和CureGN参与者的新遗传信息目前由一个 大量的核心和辅助研究。 由于CureGN将继续招募有限的一组患者，来自登记访问的血液生化标本 将需要进行处理，以便分离的DNA和RNA可以提交给NIDDK中央生物库 供科学界使用。 这项补充资金申请要求支付多达1000名患者(200名患者)的隔离费用 已经征聘，最多800人待征聘，以取代CureGN 2的需要)。 CureGN将使用Precision for Medicine的集中式服务，Precision for Medicine在以下方面有着出色的记录 精益求精的加工和质量控制标准到位。该服务提供商还将能够访问 从进入的CureGN货物到NIDDK CBR的生物样本，减少了样本转移的后勤工作 造成大量样品损失的负担和机会。

ABSTRACT Cure Glomerulonephropathy (CureGN) is a National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)-sponsored, multi-center international consortium established in 2013 to study the natural history of glomerular diseases (minimal change disease, focal segmental glomerulosclerosis, membranous nephropathy, and IgA nephropathy), understand their underlying biology, develop biomarkers to resolve disease heterogeneity, and ultimately improve therapeutic options. CureGN has made progress in: 1) establishing a large, ethnically diverse longitudinal observational cohort of glomerular disease patients; 2) creating a rich, well-curated clinical data set and linked biospecimens; 3) establishing the largest repository of digital kidney biopsies; and 4) an infrastructure that will facilitate translational and clinical research by core and ancillary study scientists. Defining the genetic and genomic landscape of the diseases studied by CureGN has been a core aim from the initiation of the study and the emerging genetic information of CureGN participants is currently utilized by a multitude of core and ancillary studies. As CureGN will continue to recruit a limited set of patients, the blood biospecimens from the enrollemtn visit will need to be processed so that isolated DNA and RNA can be submitted to the NIDDK central biorepository for use by the scientific community. This supplemental funding request asked to cover the isolation costs for up to 1000 patients (200 patients already recruited, and up to 800 to be recruited depending on the recruit to replace needs of CureGN 2). CureGN will use a centralized service by Precision for Medicine who has an outstanding track record for processing excellence and quality control metrics in place. This service provider will also be able to access the biosamples from the incoming CureGN shipments to the NIDDK CBR, reducing sample transfer logistical burden and opportunity for sample losses substantially.