Hemolytic Anemia: From Molecular Mechanisms to Therapeutic Exploitation
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 September 2025 | Viewed by 62
Special Issue Editor
Interests: sickle-cell disease (SCD); thalassaemia; RBC enzymes deficiencies; hereditary membranopathies
Special Issues, Collections and Topics in MDPI journals
Special Issue Information
Dear Colleagues,
Hemolytic anemias encompass a diverse group of disorders characterized by the premature destruction of red blood cells (RBCs), leading to reduced RBC lifespan and anemia. These conditions, such as hereditary spherocytosis, glucose-6-phosphate dehydrogenase (G6PD) deficiency, or sickle cell disease, may be inherited or acquired, as is the case for autoimmune hemolytic anemia. Advances in molecular biology have significantly enhanced our understanding of underlying pathophysiological mechanisms, revealing mutations in membrane proteins, cytoskeletal components, enzymes of the glycolytic pathway, and hemoglobin structure or synthesis. In particular, next-generation sequencing (NGS) technologies have accelerated the diagnosis of rare hereditary hemolytic anemias by uncovering novel pathogenic variants and genotype–phenotype correlations.
This molecular insight has opened new avenues for therapeutic exploitation. Targeted therapies now aim to modulate defective pathways, restore red cell homeostasis, or suppress aberrant immune responses. For instance, pharmacologic agents, such as voxelotor and L-glutamine, are shown to be beneficial for treating sickle cell disease, while gene therapy and genome editing hold promise as long-term cures. Complement inhibitors have transformed the management of paroxysmal nocturnal hemoglobinuria and atypical hemolytic uremic syndrome. Moreover, supportive treatments, including iron chelation, transfusion strategies, and bone marrow transplantation, remain essential in specific contexts.
The translational journey from molecular understanding to clinical intervention exemplifies the progress in precision medicine. Continued research is vital to expand the number of therapeutic options, especially for ultra-rare hemolytic anemias, and to ensure reasonable access to advanced diagnostics and personalized treatments across healthcare systems.
Prof. Dr. Joan-Lluis Vives-Corrons
Guest Editor
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Keywords
- hemolytic anemias
- red blood cells
- sickle cell disease
- targeted therapies
- precision medicine
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