Childhood Liver Disease Research Network (ChiLDReN) - UCSF (CC)

项目摘要/摘要 加州大学旧金山分校目前是儿童肝病研究和教育的十个原创网站之一 儿童肝病研究网络(儿童)现在称为儿童肝病研究网络(儿童)。这个 儿童的具体目标包括但不限于：1)儿童的延续 对儿童的前瞻性纵向研究继续为辅助研究提供数据和生物标本 旨在发现儿童肝病前和肝后的新诊断、病因和治疗方案 移植2)儿童肝病非侵入性标志物的识别和验证 患者包括但不限于使用FORCE FibroScan机器的新成像方式。3)第一阶段/第二阶段 治疗儿童胆汁淤积症的新药物研究。4)Alagille综合征和Alpha综合征的修饰基因研究 1-抗胰蛋白酶缺乏症5)扩大到包括其他胆汁淤积症的儿童工业和 患者权益倡导团体的支持。6)继续对小型试点和示范项目提供支持 在网络内。继续向科学和技术人员提供有关儿科肝病的教育 通过出版物和网站为社区服务。

Project Summary/Abstract UCSF is currently one of the ten original sites of the Childhood Liver Disease Research and Education Network (ChiLDREN) now referred to as the Childhood Liver Disease Research Network (ChiLDReN). The specific objectives of the ChiLDReN include but are not limited to: 1) Continuation of the ChiLDReN prospective longitudinal studies of children to continue to provide data and biospecimens for ancillary studies aimed at discovering new diagnostics, etiologic and treatment options for children both pre and post liver transplantation.2) Identification and validation of non-invasive markers of liver disease in our ChiLDReN patients including but not limited to new imaging modalities with the FORCE fibroscan machine. 3) Phase I/II study of new agents to treat cholestasis in children. 4) Study of modifier genes in Alagille Syndrome and Alpha- 1-antitrypsin deficiency 5) Expansion to include other diseases of cholestasis in children with industry and patient advocacy group support. 6) Continue to provide support for small pilot and demonstration projects within the Network.7) Continue to provide education about pediatric liver diseases to the scientific and lay communities through publications and the website.