Gene addition for beta thalassemia

JL Kwiatkowski - Annals of the New York Academy of Sciences, 2023 - Wiley Online Library
Individuals with transfusion‐dependent beta thalassemia require a high burden of care and
experience significant morbidity from the underlying disease and its treatment, which …

[HTML][HTML] Thalassemia: Pathophysiology, diagnosis, and advances in treatment

IZ Sadiq, FS Abubakar, HS Usman, AD Abdullahi… - Thalassemia …, 2024 - mdpi.com
Thalassemia represents a diverse group of inherited hematological disorders characterized
by defective globin chain synthesis, leading to chronic anemia and associated …

Thalassemia-free and graft-versus-host-free survival: outcomes of hematopoietic stem cell transplantation for thalassemia major, Turkish experience

MA Yesilipek, V Uygun, A Kupesiz, G Karasu… - Bone Marrow …, 2022 - nature.com
We report the national data on the outcomes of hematopoietic stem cell transplantation
(HSCT) for thalassemia major (TM) patients in Turkey on behalf of the Turkish Pediatric Stem …

[HTML][HTML] Hematopoietic stem cell transplantation for thalassemia major using HLA fully-matched and mismatched donor grafts

C Huang, Y Qu, S Liu, S Nie, H Jiang - Translational Pediatrics, 2021 - ncbi.nlm.nih.gov
Background Until now, allogeneic hematopoietic stem cell transplantation (allo-HSCT) is the
only effective method to cure Thalassemia major. However, it has not been determined …

Finding a balance in reduced toxicity hematopoietic stem cell transplantation for thalassemia: role of infused CD3+ cell count and immunosuppression

B Meissner, P Lang, P Bader, M Hoenig… - Bone Marrow …, 2024 - nature.com
We performed a retrospective analysis on 124 patients with transfusion-dependent
thalassemia who were registered in the German pediatric registry for stem cell …

Haploidentical transplant for paediatric patients with severe thalassaemia using post‐transplant cyclophosphamide and methotrexate: A prospectively registered …

J Hu, S Gong, K Chen, R Yang, L Wang… - British Journal of …, 2023 - Wiley Online Library
Haploidentical transplantation strategies for patients with transfusion‐dependent
thalassaemia (TD‐TM) remain to be investigated. In this study, 54 paediatric patients with TD …

[HTML][HTML] G-CSF-mobilized blood and bone marrow grafts as the source of stem cells for HLA-identical sibling transplantation in patients with thalassemia major

Q Li, J Luo, Z Zhang, L Liu, L Luo, G Yang, R Liu… - Biology of Blood and …, 2019 - Elsevier
As an inherited anemia, thalassemia major (TM) is currently only curable with allogeneic
hematopoietic stem cell transplantation (allo-HSCT). Here we report an allo-HSCT protocol …

Genome-wide methylation and gene-expression analyses in thalassemia

W Zhang, X Li, U Yu, X Huang, H Wang, Y Lu… - Aging (Albany …, 2024 - pmc.ncbi.nlm.nih.gov
Thalassemia is the most common autosomal genetic disorder in humans. The pathogenesis
of thalassemia is principally due to the deletion or mutation of globin genes that then leads …

The effect of bone marrow-derived mesenchymal stem cell co-transplantation with hematopoietic stem cells on liver fibrosis alleviation and survival in patients with …

T Rostami, A Kasaeian, N Maleki, M Nikbakht… - Stem Cell Research & …, 2021 - Springer
Background Hepatic fibrosis is a common complication in transfusion-dependent
thalassemia patients. Data on the co-transplantation of mesenchymal stem cells (MSCs) with …

Comparable outcomes of allogeneic peripheral blood versus bone marrow hematopoietic stem cell transplantation from a sibling donor for pediatric patients

BK Kim, KT Hong, JY Choi, H Kim, HJ Park… - Annals of Hematology, 2024 - Springer
Traditionally, bone marrow (BM) has been preferred as a source of stem cells (SCs) in
pediatric hematopoietic SC transplantation (HSCT); however, the use of peripheral blood …