Advances in Hematopoietic Stem Cell Research and Therapeutic Strategies for Thalassemia

Dear Colleagues,

Thalassemia is one of the most common genetic disorders globally, resulting from defects in hemoglobin production. It is associated with significant morbidity and mortality. In severe cases, regular blood transfusions combined with iron chelation therapy remain the standard treatment to manage the disease and prevent organ damage.

Allogeneic Hematopoietic Stem Cell Transplantation (Allo-HSCT) remains the only curative approach currently available for Thalassemia. However, its widespread use is limited by several challenges, including transplantation related mortality (TRM), graft vs. host disease (GvHD), graft failure, high cost, and limited availability of suitable HLA-matched related donors. Expanding the use of allo-HSCT across major histocompatibility (MHC) barriers could significantly increase donor availability. Recent advancements in Reduced Intensity Conditioning (RIC) regimens, T-cell depletion strategies, and the use of anti-3rd party central memory veto T cells capable of selectively eliminating the threat of alloreactive T and NK cells have shown promise in improving allo-HSCT outcomes. While achieving complete donor chimerism using myeloablative conditioning regimen is ideal, durable mixed chimerism can still support a functional graft in some Thalassemia patients.

Alternatively, autologous HSCs engineered to express a functional β-globin gene or edited to correct the faulty gene offer a promising approach to completely cure transfusion dependent Thalassemia (TDT) without the risk of alloreactivity.

In the proposed Special Issue, we expect the following aspects to be covered:

1. Recent advancements in allogeneic hematopoietic stem cell transplantation to cure Thalassemia.
2. Autologous hematopoietic stem cell transplantation combined with gene therapy and gene editing to correct Thalassemia.
3. Emerging insights into niche-HSC crosstalk within the Bone Marrow microenvironment to enhance the clinical outcome of HSCT.

Dr. Sandeep Yadav
Dr. Aloukick Kumar Singh
Guest Editors

Manuscript Submission Information

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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. Thalassemia Reports is an international peer-reviewed open access quarterly 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 1000 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.

• thalassemia
• allogeneic hematopoietic stem cell transplantation (allo-HSCT)
• graft vs. host disease (GvHD)
• reduced intensity conditioning (RIC)
• autologous HSCs
• gene therapy
• bone marrow niche