Therapeutic Approaches Targeting PAX3-FOXO1 and Its Regulatory and Transcriptional Pathways in Rhabdomyosarcoma.
Therapeutic Approaches Targeting PAX3-FOXO1 and Its Regulatory and Transcriptional Pathways in Rhabdomyosarcoma.
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DOI：
10.3390/molecules23112798
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发表时间：
2018-10-28
期刊：
影响因子：
--
通讯作者：
Barr FG
中科院分区：
文献类型：
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作者：
Nguyen TH;Barr FG
横纹肌肉瘤（RMS）是与骨骼肌谱系相关的软组织癌症家族，主要发生在儿童和年轻人中。一种特异性染色体易位t（2;13）（q35;q14），产生嵌合致癌转录因子PAX 3-FOXO 1，已被鉴定为侵袭性肺泡型RMS的标志。PAX 3-FOXO 1与其他分子变化合作，促进各种人类和小鼠模型中的致癌转化和肿瘤发生。它的表达通常限于RMS肿瘤细胞，因此为这些RMS肿瘤的治疗方法提供了非常特异的靶标。在这篇文章中，我们回顾了PAX 3-FOXO 1作为一种转录因子在这种癌症的发病机制的最新认识，并讨论了最近的事态发展，针对这种癌蛋白治疗RMS。
Rhabdomyosarcoma (RMS) is a family of soft tissue cancers that are related to the skeletal muscle lineage and predominantly occur in children and young adults. A specific chromosomal translocation t(2;13)(q35;q14) that gives rise to the chimeric oncogenic transcription factor PAX3-FOXO1 has been identified as a hallmark of the aggressive alveolar subtype of RMS. PAX3-FOXO1 cooperates with additional molecular changes to promote oncogenic transformation and tumorigenesis in various human and murine models. Its expression is generally restricted to RMS tumor cells, thus providing a very specific target for therapeutic approaches for these RMS tumors. In this article, we review the recent understanding of PAX3-FOXO1 as a transcription factor in the pathogenesis of this cancer and discuss recent developments to target this oncoprotein for treatment of RMS.