Results 51 to 60 of about 42,354 (173)
Lymphocyte subsets in untreated thalassemia patients: differences by genotype and age
BackgroundThe Lymphocyte subsets in treatment-naïve patients and their variations among different genetic subtypes and age groups remain poorly characterized.MethodsTo characterize the distribution of lymphocyte subsets in treatment-naïve thalassemia ...
Renbin Zhao +32 more
doaj +1 more source
Differential expression and clinical significance of miR-124-3p in β-thalassemia [PDF]
Objective To investigate the differential expression of miR-124-3p in peripheral blood and clinical significance of patients with β-thalassemia. Methods Peripheral blood samples were collected from 33 patients with β-thalassemia and 30 healthy controls
CAO Luoyuan, DONG Wenxu, YANG Jing, GUO Liwen, LU Jiaojiao, ZHENG Xian, FU Xianguo
doaj +1 more source
ABSTRACT Exagamglogene autotemcel (exa‐cel) is a one‐time, ex vivo, CRISPR‐Cas9 gene edited cell therapy approved for patients with transfusion dependent β‐thalassemia (TDT) aged 12–35 years. In a Phase 3 study (CLIMB THAL‐111), exa‐cel treatment resulted in reactivation of fetal hemoglobin and increases in total hemoglobin, leading to transfusion ...
Sujit Sheth +25 more
wiley +1 more source
Abstract Transfusion-dependent thalassemia (TDT) is a type of protein aggregation disease. Its clinical heterogeneity imposes challenges in effective management. Red blood cell (RBC) variables may be clinically relevant as mechanistic parts or tellers of TDT pathophysiology.
Konstantina Theocharaki +25 more
openaire +3 more sources
Therapeutic Silencing of Tmprss6 Reduces Iron‐Induced Inflammation and Prolongs Survival in MDS Mice
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 +13 more
wiley +1 more source
Deletional a-thalassemia 1 gene detection and hematological analysis in carrier with β-thalassemia
There are high prevalence of β-thalassemia and α-thalassemia in the upper Northern Thailand. Thus, the interaction between β-thalassemia and α-thalassemia can be occurred.
Sitthichai Panyasai
doaj
Thalassemia β major is a hereditary disorder caused by mutations in the β-globin gene, which regulates the formation of one of the components that make up hemoglobin. This disorder results in the production of β-globin chains being reduced or not formed.
Fantya Cerebella Aslamy +2 more
doaj +1 more source
β-thalassemia, a hereditary blood disorder caused by defective synthesis of hemoglobin β globin chains, leads to ineffective erythropoiesis and chronic anemia that may require blood transfusions.
Maria Domenica Cappellini +19 more
doaj +1 more source
Expanding the Utility of Exome Sequencing in Preventive and Population Genetics
ABSTRACT Carrier screening is a long‐standing genetic testing process offered to at‐risk couples, with or without a family history, who might have pregnancies affected by an autosomal recessive (AR) or X‐linked (XL) disorder. A total of 276 unrelated individuals, initially referred for rare disorder screening by clinicians, were enrolled in this study ...
Charilaos Kostoulas +6 more
wiley +1 more source
ObjectiveOur aim is to review the safety and efficacy of hydroxyurea (HU) on β-thalassemia patients.MethodsStudies that evaluated the safety and efficacy of HU on β-thalassemia patients were searched in Pub-Med, Cochrane Databases, Web of Science, China ...
Tianmin Huang +6 more
doaj +1 more source

