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Paroxysmal Nocturnal Hemoglobinuria: Pathophysiology and Novel Therapeutic Approaches

A special issue of International Journal of Molecular Sciences (ISSN 1422-0067). This special issue belongs to the section "Molecular Pathology, Diagnostics, and Therapeutics".

Deadline for manuscript submissions: 30 June 2024 | Viewed by 45

Special Issue Editor

Dr. Jun-Ichi Nishimura
E-Mail Website
Guest Editor
Department of Hematology and Oncology, Osaka University Graduate School of Medicine, Suita, Japan
Interests: hematology; bone marrow failure; hemolytic anemia; paroxysmal nocturnal hemoglobinuria; complement

Special Issue Information

Dear Colleagues,

Paroxysmal nocturnal hemoglobinuria (PNH) is a hematopoietic stem cell disorder characterized by complement-mediated intravascular hemolysis caused by the clonal expansion of hematopoietic stem cells with a mutation in a gene involved in GPI-anchor synthesis, such as PIGA. At this moment, the mechanism of PNH clonal expansion is still not fully resolved. The anti-C5 antibody eculizumab (Soliris®), a terminal complement inhibitor, was developed as a therapeutic for PNH hemolysis, and it not only markedly inhibited hemolysis and prevented thrombosis, but it also dramatically improved both quality of life and prognosis. However, to overcome the new challenge of manifesting extravascular hemolysis via complement C3 opsonization, various proximal complement inhibitors targeting C3, Factor D, and Factor B are being developed.

This Special Issue focuses on the molecular pathogenesis of PNH and therapeutic strategies for novel anticomplement agents.

Dr. Jun-Ichi Nishimura
Guest Editor

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. International Journal of Molecular Sciences is an international peer-reviewed open access semimonthly journal published by MDPI.

Please visit the Instructions for Authors page before submitting a manuscript. There is an Article Processing Charge (APC) for publication in this open access journal. For details about the APC please see here. 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.


  • paroxysmal nocturnal hemoglobinuria
  • complement
  • intravascular hemolysis
  • extravascular hemolysis
  • breakthrough hemolysis
  • terminal complement inhibitor
  • proxymal complement inhibitor

Published Papers

This special issue is now open for submission.
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