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Biomolecules
Special Issues
Molecular Mechanisms and Therapeutic Targets in Leukaemia
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Molecular Mechanisms and Therapeutic Targets in Leukaemia

A special issue of Biomolecules (ISSN 2218-273X). This special issue belongs to the section "Molecular Medicine".

Deadline for manuscript submissions: 30 September 2025 | Viewed by 1368

Special Issue Editors

Dr. Sabrina Tosi

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Guest Editor
Leukaemia and Chromosome Research Laboratory, Department of Life Sciences, Brunel University London, Kingston Ln, Uxbridge, London UB8 3PH, UK
Interests: chromosomal rearrangements; molecular cytogenetics; genome organization; leukaemiaa
Special Issues, Collections and Topics in MDPI journals
Dr. Denise Ragusa

E-Mail Website
Guest Editor
Department of Life Sciences, Brunel University London, Kingston Ln, Uxbridge, London UB8 3PH, UK
Interests: chromosome biology and the transcriptomics of rare leukaemia subtypes, through the development of in vitro models; 2D–3D cell cultures; gene editing (CRISPR/Cas9); molecular cytogenetic methods; bioinformatics of omics data
Special Issues, Collections and Topics in MDPI journals

Special Issue Information

Dear Colleagues,

This Special Issue on Molecular Mechanisms and Therapeutic Targets in Leukaemia aims to provide a comprehensive platform for the latest advancements and insights into the molecular mechanisms at the basis of leukaemia and the identification of potential targets for therapy. By bringing together diverse perspectives and research findings, this Special Issue seeks to enhance our understanding of leukaemia initiation and progression, fostering the development of precision medicine approaches for this challenging disease. Researchers, clinicians, and experts in the field are encouraged to contribute their valuable insights to advance our knowledge and improve patient outcomes in leukaemia.

This Special Issue invites original research articles, reviews, and communications encompassing a wide range of topics, including, but not limited to, the following:

  • Molecular profiling of leukaemia subtypes and their genetic drivers.
  • Identification and characterization of genetic mutations, chromosomal rearrangements, and copy number variations associated with different leukaemia types.
  • Functional genomics and pathway analyses to elucidate the molecular mechanisms underlying leukaemia development and progression.
  • Genomic instability and its impact on leukaemia evolution and treatment resistance.
  • Role of epigenetic modifications in leukaemogenesis and their potential as therapeutic targets.
  • Applications of next-generation sequencing, single-cell genomics, and other cutting-edge technologies in leukaemia research.
  • Genomic biomarkers for the risk stratification, treatment selection, and monitoring of leukaemia patients.

Dr. Sabrina Tosi
Dr. Denise Ragusa
Guest Editors

Manuscript Submission Information

Manuscripts should be submitted online at www.mdpi.com by registering and logging in to this website. Once you are registered, click here to go to the submission form. Manuscripts can be submitted until the deadline. All submissions that pass pre-check are peer-reviewed. Accepted papers will be published continuously in the journal (as soon as accepted) and will be listed together on the special issue website. Research articles, review articles as well as short communications are invited. For planned papers, a title and short abstract (about 100 words) can be sent to the Editorial Office for announcement on this website.

Submitted manuscripts should not have been published previously, nor be under consideration for publication elsewhere (except conference proceedings papers). All manuscripts are thoroughly refereed through a single-blind peer-review process. A guide for authors and other relevant information for submission of manuscripts is available on the Instructions for Authors page. Biomolecules is an international peer-reviewed open access monthly journal published by MDPI.

Please visit the Instructions for Authors page before submitting a manuscript. The Article Processing Charge (APC) for publication in this open access journal is 2700 CHF (Swiss Francs). Submitted papers should be well formatted and use good English. Authors may use MDPI's English editing service prior to publication or during author revisions.

Keywords

  • leukaemia
  • blood
  • genetics
  • genomics
  • chromosome

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Published Papers (1 paper)

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Review

29 pages, 759 KiB  
Review
XPO1/Exportin-1 in Acute Myelogenous Leukemia; Biology and Therapeutic Targeting
by Øystein Bruserud, Frode Selheim, Maria Hernandez-Valladares and Håkon Reikvam
Biomolecules 2025, 15(2), 175; https://doi.org/10.3390/biom15020175 - 24 Jan 2025
Viewed by 1043
Abstract
Exportin 1 is responsible for the export of hundreds of proteins, several RNA species and ribosomal components from the nucleus to the cytoplasm. Several transported proteins are important for regulation of cell proliferation and survival both in normal and malignant cells. We review [...] Read more.
Exportin 1 is responsible for the export of hundreds of proteins, several RNA species and ribosomal components from the nucleus to the cytoplasm. Several transported proteins are important for regulation of cell proliferation and survival both in normal and malignant cells. We review the biological importance and the possibility of therapeutic targeting of Exportin 1 in acute myeloid leukemia (AML). Exportin 1 levels can be increased in human primary AML cells, and even exportin inhibition as monotherapy seems to have an antileukemic effect. The results from Phase I/II studies also suggest that exportin inhibition can be combined with conventional chemotherapy, including intensive induction and consolidation therapy possibly followed by allogeneic stem cell transplantation as well as AML-stabilizing therapy in elderly/unfit patients with hypomethylating agents. However, the risk of severe toxicity needs to be further evaluated; hematological toxicity is common together with constitutional side effects, electrolyte disturbances, and gastrointestinal toxicity. A recent randomized study of intensive chemotherapy with and without the Exportin inhibitor selinexor in elderly patients showed reduced survival in the selinexor arm; this was due to a high frequency of relapse and severe infections during neutropenia. Experimental studies suggest that Exportin 1 inhibition can be combined with other forms of targeted therapy. Thus, Exportin 1 inhibition should still be regarded as a promising strategy for AML treatment, but future studies should focus on the risk of toxicity when combined with conventional chemotherapy, especially in elderly/unfit patients, combinations with targeted therapies, identification of patient subsets (AML is a heterogeneous disease) with high susceptibility, and the possible use of less toxic next-generation Exportin 1 inhibitors. Full article
(This article belongs to the Special Issue Molecular Mechanisms and Therapeutic Targets in Leukaemia)
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