Results 101 to 110 of about 140,103 (295)

Gene Editing for Haemophilia—The Next Frontier

open access: yesHaemophilia, 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   +3 more
wiley   +1 more source

Enhanced induction of fetal hemoglobin by the combination of decitabine with RN-1 in β-thalassemia/HbE erythroid progenitor cells

open access: yesMolecular Medicine
Background Fetal hemoglobin (HbF; α2γ2) induction is a well-established approach for β-hemoglobinopathies, including sickle cell disease (SCD) and β-thalassemia.
Tiwaporn Nualkaew   +9 more
doaj   +1 more source

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

open access: yesHemodialysis 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   +6 more
wiley   +1 more source

Luspatercept in Transfusion-Dependent Thalassemia With Red Blood Cell Antibodies: A Case Series. [PDF]

open access: yesEJHaem
ABSTRACT Red blood cell (RBC) antibodies complicate transfusion support in transfusion‐dependent thalassemia (TDT), and data on luspatercept in this setting remain limited. We retrospectively reviewed seven adults with TDT and RBC antibodies treated with luspatercept at Srinagarind Hospital from January 2025 to January 2026.
Teawtrakul N.
europepmc   +2 more sources

Hair‐on‐end sign in severe sickle cell disease

open access: yes
British Journal of Haematology, EarlyView.
Raquel da Costa Neves   +2 more
wiley   +1 more source

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

open access: yesInternational 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   +9 more
wiley   +1 more source

Iron Overload: Pathophysiology, Diagnosis and Monitoring

open access: yesInternational Journal of Laboratory Hematology, EarlyView.
ABSTRACT Iron overload is associated with significant health risks, underscoring the importance of understanding its pathophysiology as well as establishing accurate diagnostic and monitoring methods. Chronic iron overload is associated with either genetic disorders characterized by excessive iron accumulation (hereditary hemochromatosis), or is ...
Elena Chatzikalil   +3 more
wiley   +1 more source

Home - About - Disclaimer - Privacy