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Open access Sep 2026

Contemporary Management of Thalassemia: A Perspective on Current Standards and the Emergence of FDA‐Approved Oral Therapy

Mitapivat represents a significant advance in thalassemia management by targeting erythrocyte metabolism and providing the first FDA‐approved oral treatment for anemia in adults with α or β thalassemia.

Fnu Zainab, Abdul Haseeb Hasan, Muhammad Hafi Abid et al. · 0 citations
Review Open access Sep 2026

Sickle Cell Anemia: Therapeutic Advances, Disease-Modifying Therapies, and Future Prospects

The evidence demonstrates that advances in disease-modifying therapies and potentially curative strategies are transforming the management of sickle cell disease, reinforcing the need for long-term studies and public policies that expand access to innovative technologies.

Rosane Rezende de Souza Giuliani, Felipe Santos Teixeira Martiniano, Nicole Mioto Medeiros et al. · 0 citations
Open access Sep 2026

An Integrated Gene Therapy Strategy for γ-Globin Addition and HbF Reactivation in β-Thalassemia

Background: β-Thalassemia and other β-hemoglobinopathies arise from defective β-globin production, and reactivating fetal hemoglobin (HbF) is a well-established strategy to ameliorate disease severity. Two principal gene-therapy approaches—erythroid-specific γ-globin gene addition and shRNA/shmiRNA-mediated knockdown o...

K. Paschoudi, Ninos-Ioannis Vasiloudis, Fotios Papadopoulos et al. · 0 citations
#gene editing Review Open access Sep 2026

Advances in Hematopoietic Stem Cell Transplantation: A Review of Recent Developments and Challenges

This review synthesizes contemporary breakthroughs in HSCT biology, technology, and clinical application, critically examines unresolved barriers, and highlights future directions, including Artificial Intelligence (AI)-driven predictive analytics, universal donor platforms, and equitable global access strategies.

M. Abah, Ovwigho Ugbede, O. C. et al. · 0 citations
#gene editing Review Oct 2026

The expanding therapeutic landscape of β-thalassaemia.

Advances in β-thalassaemia treatment over the past decade are discussed, including newly approved and late-stage therapies and their future implications, and remaining research gaps are highlighted.

K. Musallam, Sujit Sheth, Franco Locatelli et al. · 2 citations
#gene editing Review Open access Oct 2026

Precision Medicine in Sickle Cell Disease: From Molecular Pathways to Curative Strategies.

Sickle cell disease (SCD) is a monogenic β-globin disorder characterized by hemoglobin S polymerization, hemolysis, and vaso-occlusion, yet it exhibits remarkable clinical heterogeneity. Despite advances in supportive care and disease-modifying therapies, substantial variability in severity, treatment response, and lon...

Dina Tluli, Omar Tluli, Abdelrahman A. Karen et al. · 0 citations

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