Results 71 to 80 of about 36,735 (203)

Gene Editing for Haemophilia—The Next Frontier

Haemophilia, EarlyView.
ABSTRACT The recently approved haemophilia A and B gene therapies via adeno‐associated virus (AAV) showed a promising therapeutic response after a single injection, but there are still limitations, including the potential loss of transgene expression and restriction in adults.
Mirko Pinotti, Gregory A. Newby, Sundar Selvaraj, Steven W. Pipe   +3 more
wiley   +1 more source

Safety and Efficacy of Iron Chelators in Patients With Chronic Kidney Disease: A Systematic Review and Meta‐Analysis

Hemodialysis International, EarlyView.
ABSTRACT Background Iron overload in chronic kidney disease contributes to oxidative injury. The role of iron chelators, standard in transfusion‐related overload, remains unclear in chronic kidney disease. Aim To evaluate the efficacy and safety of iron chelators in adults with chronic kidney disease.
Humam Emad Rajha, Richard Shamoon, Ameer Almeshagbeh, Samah Masry, Fadel Alqatati, Baha H. Abuajameia, Abdulqadir J. Nashwan   +6 more
wiley   +1 more source

Optimal Hypoxia Mimetic Small Molecules for Enhancing Angiogenic Properties of Stem Cells From Human Exfoliated Deciduous Teeth

International Endodontic Journal, EarlyView.
ABSTRACT Aim Hypoxic preconditioning of cells holds promise for regenerative therapies, yet identifying effective and safe methods for clinical application remains challenging. We aimed to determine optimal hypoxia‐mimetic small molecules (SMs) that stabilize hypoxia‐inducible factor‐1α (HIF‐1α) and their dosages for hypoxic preconditioning in stem ...
Hong Wang, Jiawei Liu, Xintao Yang, Mohamad Koohi‐Moghadam, Shuting Gao, Yuanyuan Han, Dineshi Sewvandi Thalakiriyawa, James Kit Hon Tsoi, Yanqi Yang, Waruna Lakmal Dissanayaka   +9 more
wiley   +1 more source

FREQUENCY OF BETA THALASSEMIA TRAIT AMONG THE HEALTHY INDIVIDUALS - A SINGLE CENTRE STUDY

Pakistan Armed Forces Medical Journal, 2018
Objective: To determine the frequency of beta thalassemia trait among the asymptomatic healthy individuals. Study Design: Cross sectional study. Place and Duration of Study: This was carried out at Pakistan Air Force (PAF) Hospital Lahore from ...
Muhammad Arif Sadiq, Ahmad Muqeem, Rizwan Yusuf, Asma Bilal   +3 more
doaj   +4 more sources

Iron dysregulation in beta-thalassemia

Asian Pacific Journal of Tropical Medicine, 2016
Iron deficiency anemia and iron overload conditions affect more than one billion people worldwide. Iron homeostasis involves the regulation of cells that export iron into the plasma and cells that utilize or store iron. The cellular iron balance in humans is primarily mediated by the hepcidin-ferroportin axis.
Leecharoenkiat, Kamonlak, Lithanatudom, Pathrapol, Sornjai, Wannapa, Smith, Duncan R.   +3 more
openaire   +2 more sources

A retrospective analysis of the antigen‐negative red blood cell supply conducted at a single centre in China

Transfusion Medicine, EarlyView.
Abstract Objective This study aimed to analyse the distribution and demand patterns of antigen‐negative red blood cells (RBCs) in Shandong Province, China (2022–2024), with a focus on ABO blood groups and clinically significant antigens. The research was designed to provide rigorous data for advancing precision transfusion protocols and to establish a ...
Aiping Zhao, Xiujiao Jiang, Li An, Yuanfeng Chen, Weidong He, Qun Xu, Wenchao Li, Yingfang Pan   +7 more
wiley   +1 more source

A global survey of blood transfusion practices for patients with sickle cell disease

Transfusion, EarlyView.
Abstract Background Sickle cell disease (SCD) affects over 7 million people globally, with blood transfusion remaining a cornerstone of management. However, contemporary transfusion practices across diverse settings remain poorly characterized. We evaluated global transfusion practices for patients with SCD to identify gaps and inform resource ...
Jeremy W. Jacobs, Arwa Z. Al‐Riyami, Jeannie Callum, Meghan Delaney, David Daniel, Jenny White, Christopher Bocquet, Mohammed Farouk, Claude Tayou, Chinelo P. Onyenekwu, Kristina Prus, Divjot Singh Lamba, Julie Makani, Quentin Eichbaum, Luiz Amorim, Silvano Wendel, France Pirenne, Vernon Louw, Evan M. Bloch   +18 more
wiley   +1 more source

Therapeutic Silencing of Tmprss6 Reduces Iron‐Induced Inflammation and Prolongs Survival in MDS Mice

American Journal of Hematology, Volume 101, Issue 9, Page 2105-2122, September 2026.
ABSTRACT Myelodysplastic syndromes (MDS) are a heterogeneous group of clonal hematopoietic disorders characterized by ineffective hematopoiesis, cytopenias, and an increased risk of progression to acute myeloid leukemia (AML). Despite advances in supportive and targeted therapies, disease‐modifying interventions remain limited.
Shahla Vilcassim, Nuttanan Pholngam, Rattanawan Thubthed, Tiwaporn Nualkaew, Saovaros Svasti, Pornthip Chaichompoo, Kai Kysenius, Peter J. Crouch, Sibylle Dames, Mona Eisermann, Alberto Martinez, Ute Schaeper, Jim Vadolas, George Grigoriadis   +13 more
wiley   +1 more source

Myelodysplastic Syndromes: 2026 Update on Diagnosis, Risk‐Stratification and Management

American Journal of Hematology, Volume 101, Issue 9, Page 2393-2411, September 2026.
ABSTRACT Disease Overview The myelodysplastic syndromes (MDS) are a heterogeneous group of myeloid disorders characterized by peripheral blood cytopenias and increased risk of transformation to acute myelogenous leukemia (AML). MDS occurs more frequently in older males and in individuals with prior exposure to cytotoxic therapy.
Guillermo Garcia‐Manero
wiley   +1 more source

Beta Thalassemia [PDF]

, 2020
  +4 more sources