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慢性淋巴细胞白血病中的微小RNA:预后及靶向治疗的奇迹还是幻影?

MicroRNAs in chronic lymphocytic leukemia: miRacle or miRage for prognosis and targeted therapies?

作者信息

Van Roosbroeck Katrien, Calin George A

机构信息

Department of Experimental Therapeutics, The University of Texas MD Anderson Cancer Center, Houston, TX.

Department of Experimental Therapeutics, The University of Texas MD Anderson Cancer Center, Houston, TX; Center for RNA Interference and Non-Coding RNAs, The University of Texas MD Anderson Cancer Center, Houston, TX; Department of Leukemia, The University of Texas MD Anderson Cancer Center, Houston, TX.

出版信息

Semin Oncol. 2016 Apr;43(2):209-14. doi: 10.1053/j.seminoncol.2016.02.015. Epub 2016 Feb 8.

Abstract

Chronic lymphocytic leukemia (CLL) is a heterogeneous disease and has a highly variable clinical course with survival ranging from a couple of months to several decades. MicroRNAs (miRNAs), small non-coding RNAs that regulate transcription and translation of genes, have been found to be involved in CLL initiation, progression, and resistance to therapy. In addition, they can be used as prognostic biomarkers and as targets for novel therapies. In this review, we describe the association between miRNAs and the cytogenetic aberrations commonly found in CLL, as well as with other prognostic factors. We describe the presence of miRNAs as extracellular entities in the plasma and serum of CLL patients and discuss their role in resistance to therapy. Finally, we will explore the potential of targeted miRNA therapy for the treatment of CLL, with a special emphasis on MRX34, the first miRNA mimic that is currently being evaluated for clinical use.

摘要

慢性淋巴细胞白血病(CLL)是一种异质性疾病,临床病程高度可变,生存期从几个月到几十年不等。微小RNA(miRNA)是一类调节基因转录和翻译的小型非编码RNA,已发现其参与CLL的起始、进展及对治疗的抵抗。此外,它们可用作预后生物标志物及新型治疗的靶点。在本综述中,我们描述了miRNA与CLL中常见的细胞遗传学异常以及其他预后因素之间的关联。我们阐述了miRNA作为细胞外实体在CLL患者血浆和血清中的存在情况,并讨论了它们在治疗抵抗中的作用。最后,我们将探讨靶向miRNA治疗CLL的潜力,特别强调MRX34,这是首个目前正在进行临床评估的miRNA模拟物。

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