[HTML][HTML] Fighting rare cancers: lessons from fibrolamellar hepatocellular carcinoma

SM Simon - Nature Reviews Cancer, 2023 - nature.com
The fight against rare cancers faces myriad challenges, including missed or wrong
diagnoses, lack of information and diagnostic tools, too few samples and too little funding …

Proteolysis-targeting chimeras in biotherapeutics: Current trends and future applications

Q Li, L Zhou, S Qin, Z Huang, B Li, R Liu… - European Journal of …, 2023 - Elsevier
The success of inhibitor-based therapeutics is largely constrained by the acquisition of
therapeutic resistance, which is partially driven by the undruggable proteome. The …

Disruption of proteome by an oncogenic fusion kinase alters metabolism in fibrolamellar hepatocellular carcinoma

SN Levin, MD Tomasini, J Knox, M Shirani, B Shebl… - Science …, 2023 - science.org
Fibrolamellar hepatocellular carcinoma (FLC) is a usually lethal primary liver cancer driven
by a somatic dysregulation of protein kinase A. We show that the proteome of FLC tumors is …

State of the art and perspectives in pediatric hepatocellular carcinoma

G Digiacomo, RP Serra, E Turrini, A Tiri… - Biochemical …, 2023 - Elsevier
Hepatoblastoma (HB) and pediatric hepatocellular carcinoma (HCC) are rare primary
malignant liver cancers in children and young adults. HB is the most common and accounts …

Current advances in the treatment of fibrolamellar carcinoma of liver

J Gummadi, X Wang, C Xie - Journal of Hepatocellular Carcinoma, 2023 - Taylor & Francis
Fibrolamellar carcinoma (FLC) of the liver is a rare type of liver cancer that is prevalent in
children and young adults, often less than 40 years old. The etiology is unclear. It presents …

Children's Oncology Group's 2023 blueprint for research: Liver tumors

AF O'Neill, RL Meyers, HM Katzenstein… - Pediatric Blood & …, 2023 - Wiley Online Library
Liver tumors account for approximately 1%–2% of all pediatric malignancies, with the two
most common tumors being hepatoblastoma (HB) and hepatocellular carcinoma (HCC) …

[HTML][HTML] GalNAc-conjugated siRNA targeting the DNAJB1-PRKACA fusion junction in fibrolamellar hepatocellular carcinoma

C Neumayer, D Ng, D Requena, CS Jiang, A Qureshi… - Molecular Therapy, 2024 - cell.com
Fibrolamellar hepatocellular carcinoma (FLC) is a rare liver cancer caused by a dominant
recurrent fusion of the heat shock protein (DNAJB1) and the catalytic subunit of protein …

[HTML][HTML] FusionVAC22_01: a phase I clinical trial evaluating a DNAJB1-PRKACA fusion transcript-based peptide vaccine combined with immune checkpoint inhibition …

C Hackenbruch, J Bauer, JS Heitmann… - Frontiers in …, 2024 - frontiersin.org
The DNAJB1-PRKACA fusion transcript was identified as the oncogenic driver of tumor
pathogenesis in fibrolamellar hepatocellular carcinoma (FL-HCC), also known as …

A preclinical “magic bullet” against fibrolamellar hepatocellular carcinoma?

C Ducrot, M Piffoux - Molecular Therapy, 2024 - cell.com
Fibrolamellar hepatocellular carcinoma (FL-HCC) is a very rare liver cancer
(incidence±0.02/100,000, 1% HCC) that mostly affects adolescents and young adults …

[HTML][HTML] 肿瘤靶向抑制剂的PROTACs设计及在抗肿瘤新药开发中的应用*

李婧绮, 吴定宇, 谭睿 - 中国生物工程杂志, 2024 - manu60.magtech.com.cn
蛋白质的非正常表达往往会直接或间接导致癌症的发生. 目前, 靶向特定蛋白质的小分子抑制剂
在肿瘤治疗领域中得到了广泛使用. 然而, 小分子抑制剂存在易使机体产生耐药性 …