Mithramycin encapsulated in polymeric micelles by microfluidic technology as novel therapeutic protocol for beta-thalassemia [PDF]
, 2012 This report shows that the DNA-binding drug, mithramycin, can be efficiently encapsulated in polymeric micelles (PM-MTH), based on Pluronic® block copolymers, by a new microfluidic approach.
Brognara, Eleonora +8 more
core +2 more sources
Identification of Delta-beta Thalassemia in a Family with Elevated Hb F: A Case Report [PDF]
Journal of Krishna Institute of Medical Sciences University, 2020 Delta-beta Thalassemia is a rare variant of thalassemia with elevated Hb F. Heterozygous and homozygous state of delta-beta thalassemia present with features similar to Hereditary Persistence of Fetal Haemoglobin (HPFH) and beta thalassemia ...
Varun Kumar Singh +3 more
doaj
Iraqi Journal of Hematology, 2022 BACKGROUND: Anemia of β thalassemia results from a combination of ineffective erythropoiesis and hemolysis. This stimulates erythropoietin (EPO) production, which causes expansion of the bone marrow and may lead to serious deformities of the skull and ...
May Hikmat Yousif +1 more
doaj +1 more source
Transcriptional regulation of the ABCC6 gene and the background of impaired function of missense disease-causing mutations. [PDF]
, 2013 The human ATP-binding cassette family C member 6 (ABCC6) gene encodes an ABC transporter protein expressed primarily in the liver and to a lesser extent in the kidneys and the intestines.
Arányi, Tamás +5 more
core +1 more source
Efficacy of lactoferrin oral administration in the treatment of anemia and anemia of inflammation in pregnant and non-pregnant women: an interventional study [PDF]
, 2018 The discovery of the ferroportin-hepcidin complex has led to a critical review on the treatment of anemia and anemia of inflammation (AI). Ferroportin, the only known mammalian iron exporter from cells to blood, is negatively regulated by hepcidin, a ...
Antimo Cutone +6 more
core +2 more sources
Beta-thalassemia: renal complications and mechanisms: a narrative review
Hematology, 2019 Objectives: Beta-thalassemias are a group of recessively autosomal inherited disorders of hemoglobin synthesis, which, due to mutations of the beta-globin gene, lead to various degrees of defective beta-chain production, an imbalance in alpha/beta-globin
C. Demosthenous +8 more
semanticscholar +1 more source
Gene Therapy For Beta-Thalassemia: Updated Perspectives
The Application of Clinical Genetics, 2019 Allogeneic hematopoietic stem cell transplantation was until very recently, the only permanent curative option available for patients suffering from transfusion-dependent beta-thalassemia.
Garyfalia Karponi, Nikolaos Zogas
semanticscholar +1 more source
The human ankyrin 1 promoter insulator sustains gene expression in a β-globin lentiviral vector in hematopoietic stem cells. [PDF]
, 2015 Lentiviral vectors designed for the treatment of the hemoglobinopathies require the inclusion of regulatory and strong enhancer elements to achieve sufficient expression of the β-globin transgene.
Baldwin, Kismet M +12 more
core +4 more sources
Hospitalization causes due to iron overload in beta-Thalassemia in Gorgan, Iran [PDF]
, 2011 Objective: To evaluate causes of hospitalization (due to complications of iron overload and other causes) in beta-Thalassemic patients. Methodology: This study was performed on 244 patients with major beta-Thalassemia admitted in Taleghani hospital of ...
Jahazi, A. +2 more
core
Pediatric Blood &Cancer, EarlyView.ABSTRACT Background We describe clinical and biologic characteristics of neuroblastoma in older children, adolescents, and young adults (OCAYA); describe survival outcomes in the post‐immunotherapy era; and identify if there is an age cut‐off that best discriminates outcomes.
Rebecca J. Deyell +14 more
wiley +1 more source