Biology of childhood hepatoblastoma and the search for novel treatments

Q1 Biochemistry, Genetics and Molecular Biology

Advances in biological regulation Pub Date : 2024-01-01 DOI:10.1016/j.jbior.2023.100997

Marjut Pihlajoki , Katja Eloranta , Ruth Nousiainen , Ville Väyrynen , Tea Soini , Antti Kyrönlahti , Seppo Parkkila , Jukka Kanerva , David B. Wilson , Mikko P. Pakarinen , Markku Heikinheimo

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Abstract

Our research laboratory has a longstanding interest in developmental disorders and embryonic tumors, and recent efforts have focused on the pathogenesis of pediatric liver tumors. This review focuses on hepatoblastoma (HB), the most common pediatric liver malignancy. Despite advances in treatment, patients with metastatic HB have a poor prognosis, and survivors often have permanent side effects attributable to chemotherapy. In an effort to improve survival and lessen long-term complications of HB, we have searched for novel molecular vulnerabilities using a combination of patient derived cell lines, metabolomics, and RNA sequencing of human samples at diagnosis and follow-up. These studies have shed light on pathogenesis and identified putative targets for future therapies in children with advanced HB.

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儿童肝母细胞瘤生物学及新型治疗方法的探索

我们的研究实验室长期关注发育障碍和胚胎肿瘤，最近的研究重点是小儿肝脏肿瘤的发病机制。本综述的重点是肝母细胞瘤（HB），它是最常见的小儿肝脏恶性肿瘤。尽管治疗手段不断进步，但转移性肝母细胞瘤患者的预后仍然很差，幸存者往往会因化疗而产生永久性副作用。为了提高HB患者的生存率并减少其长期并发症，我们结合患者衍生细胞系、代谢组学以及诊断和随访时人体样本的RNA测序，寻找新的分子漏洞。这些研究揭示了晚期 HB 儿童的发病机制，并确定了未来疗法的潜在靶点。

本文章由计算机程序翻译，如有差异，请以英文原文为准。

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来源期刊

Advances in biological regulation Biochemistry, Genetics and Molecular Biology-Molecular Medicine

CiteScore

8.90

自引率

0.00%

发文量

审稿时长

17 days