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Review
. 2020 Feb 3:11:2040620719899818.
doi: 10.1177/2040620719899818. eCollection 2020.

Nucleophosmin1 (NPM1) abnormality in hematologic malignancies, and therapeutic targeting of mutant NPM1 in acute myeloid leukemia

Yingyu Chen  1 , Jianda Hu  2
Affiliations
Review

Nucleophosmin1 (NPM1) abnormality in hematologic malignancies, and therapeutic targeting of mutant NPM1 in acute myeloid leukemia

Yingyu Chen et al. Ther Adv Hematol. .

Abstract

Nucleophosmin (NPM1) is an abundant nucleolar protein that is implicated in a variety of biological processes and in the pathogenesis of several human malignancies. For hematologic malignancies, approximately one-third of anaplastic large-cell non-Hodgkin's lymphomas were found to express a fusion between NPM1 and the catalytic domain of anaplastic lymphoma receptor tyrosine kinase. About 50-60% of acute myeloid leukemia patients with normal karyotype carry NPM1 mutations, which are characterized by cytoplasmic dislocation of the NPM1 protein. Nevertheless, NPM1 is overexpressed in various hematologic and solid tumor malignancies. NPM1 overexpression is considered a prognostic marker of recurrence and progression of cancer. Thus, NPM1 abnormalities play a critical role in several types of hematologic malignancies. This has led to intense interest in the development of an NPM1 targeting strategy for cancer therapy. The aim of this review is to summarize present knowledge on NPM1 origin, pathogenesis, and therapeutic interventions in hematologic malignancies.

Keywords: Nucleophosmin1; hematologic malignancy; mutation; overexpression; therapy.

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Conflict of interest statement

Conflict of interest statement: The author(s) declare no conflicts of interest in preparing this article.

Figures

Figure 1.
Figure 1.
The cellular distribution of wild type and mutant NPM1 in a healthy cell and a leukemia cell. NPM1, Nucleophosmin.
See this image and copyright information in PMC

References

    1. Falini B, Mecucci C, Tiacci E, et al. Cytoplasmic nucleophosmin in acute myelogenous leukemia with a normal karyotype. N Engl J Med 2005; 352: 254–266. - PubMed
    1. Borer RA, Lehner CF, Eppenberger HM, et al. Major nucleolar proteins shuttle between nucleus and cytoplasm. Cell 1989; 56: 379–390. - PubMed
    1. Colombo E, Alcalay M, Pelicci PG. Nucleophosmin and its complex network: a possible therapeutic target in hematological diseases. Oncogene 2011; 30: 2595–2609. - PubMed
    1. Yung BY, Busch H, Chan PK. Translocation of nucleolar phosphoprotein B23 (37 kDa/pI 5.1) induced by selective inhibitors of ribosome synthesis. Biochim Biophys Acta 1985; 826: 167–173. - PubMed
    1. Papaemmanuil E, Dohner H, Campbell PJ. Genomic classification in acute myeloid leukemia. N Engl J Med 2016; 375: 900–901. - PubMed

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