Project 1: Fibrolamellar HCC

项目1：纤维板层肝癌

• 批准号: 10468829
• 负责人: MICHAEL S TORBENSON
• 金额: $ 42.55万
• 依托单位: MAYO CLINIC ROCHESTER
• 依托单位国家: 美国
• 财政年份: 2018
• 资助国家: 美国
• 起止时间: 2018-09-10 至 2024-08-31
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/10468829
• 关键词: Affect; Antibodies; Area; Binding; Biological Assay; Biopsy; Biopsy Specimen; Blood Tests; Catalytic Domain; Cells; Child; Chimera organism; Chimeric Proteins; Chromosome 19; Clinic; Clinical; Cyclic AMP-Dependent Protein Kinases; DNA; Diagnosis; Diagnostic; Diagnostic tests; Disease; Double-Blind Method; Early Diagnosis; Excision; Fibrolamellar Hepatocellular Carcinoma; Fluorescent in Situ Hybridization; Genes; Genetic; Genetically Engineered Mouse; Goals; Growth; Heat shock proteins; Hepatobiliary; Hepatocyte; Histologic; Histology; Human; Immunohistochemistry; Institution; Light; Link; Liver neoplasms; Malignant Childhood Neoplasm; Malignant Neoplasms; Messenger RNA; MicroRNAs; Molecular Conformation; Molecular Diagnosis; Morphology; Normal tissue morphology; Operative Surgical Procedures; PRKACA gene; Pathogenesis; Pathologist; Pathology; Patient Care; Patients; Pharmaceutical Preparations; Phosphotransferases; Prognosis; Proteins; RNA; Recurrence; Sampling; Sensitivity and Specificity; Symptoms; Systemic Therapy; Testing; Therapeutic; Tissues; Validation; Work; base; chimeric gene; comparative; curative treatments; human genome sequencing; improved; molecular dynamics; patient derived xenograft model; screening; small molecule; small molecule libraries; success; therapeutic target; treatment response; tumor; virtual; whole genome; young adult

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.

项目1 - 项目摘要 纤维状肝细胞癌（FLHCC）是一种致死性疾病，影响了小学儿童， 年轻人。由于缺乏现有的诊断测试和全身性，预后不佳会加剧 治疗。我们最近证明，一个染色体副本中有一个一致的删除 19在FLHCC患者中，导致嵌合基因。该嵌合体编码融合蛋白，一种组成型 蛋白激酶A的主动催化亚基A具有两个目的：（1）开发第一个诊断测试 这种癌症和（2）为这种癌症开发了驱动因素的第一个阻滞剂。