Project 1: Fibrolamellar HCC

项目1 -项目概要 纤维板层型肝细胞癌（FLHCC）是一种常见的致死性疾病，主要影响儿童， 年轻人由于缺乏现有的诊断测试和系统性检查， 疗法我们最近已经证明，在一个染色体拷贝中存在单一的、一致的缺失， 19例FLHCC患者，导致嵌合基因。这种嵌合体编码一种融合蛋白， 蛋白激酶A的活性催化亚基。该项目有两个目标：（1）开发第一个诊断测试， 这种癌症和（2）开发这种癌症的驱动程序的第一个阻断剂。

PROJECT 1 – PROJECT SUMMARY Fibrolamellar hepatocellular carcinoma (FLHCC) is an often-lethal disease affecting primarily children and young adults. The poor prognosis is exacerbated by the lack of both existing diagnostic tests and systemic therapy. We have recently demonstrated that there is a single, consistent deletion in one copy of chromosome 19 in FLHCC patients that results in a chimeric gene. This chimera encodes a fusion protein, a constitutively active catalytic subunit of protein kinase A. This project has two aims: (1) develop the first diagnostic tests for this cancer and (2) develop the first blockers for the driver for this cancer.