Project 1: Fibrolamellar HCC

项目1：纤维板层肝癌

• 批准号: 10006079
• 负责人: MICHAEL S TORBENSON
• 金额: $ 39.64万
• 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 2023-08-31
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/10006079
• 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; Proteins; RNA; Recurrence; Sampling; Sensitivity and Specificity; Symptoms; Systemic Therapy; Testing; Therapeutic; Tissues; Validation; Work; Xenograft procedure; base; chimeric gene; comparative; curative treatments; human genome sequencing; improved; molecular dynamics; outcome forecast; 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--项目总结 纤维板层状肝细胞癌是一种通常致命的疾病，主要影响儿童和 年轻人。由于缺乏现有的诊断测试和全身性检查，预后不良的情况更加严重。 心理治疗。我们最近已经证明，在一个染色体拷贝中有一个单一的、一致的缺失 19在肝癌患者中，这会导致嵌合基因。这种嵌合体编码一种融合蛋白，一种结构性的 这个项目有两个目标：(1)开发第一个诊断试验 这种癌症和(2)为这种癌症的驱动因素开发出第一种阻滞剂。