Hydroxyurea (HU) enhances fetal hemoglobin (Hb) production. An increase in total Hb level has been repeatedly reported during HU treatment in patients with sickle cell disease and in several patients with beta-thalassemia intermedia. Effects in patients with beta-thalassemia major are controversial. We now report a marked elevation of total Hb levels with HU that permitted regular transfusions ...

background: thalassemia syndromes are the most prevalent single gene disorders in iran. this study aimed to evaluate the effect of different types of beta-globin gene mutations, co-inheritance of alpha-globin gene mutations and/or xmn1 snp on disease phenotype in a large cohort of iranian patients. subjects and methods: in total, 433 patients were clinically classified into β-thalassemia major ...

Background Thalassemia is the most common hereditary disorder worldwide. The patient's’ survival is dependent on lifetime blood transfusion which leads to iron overload and its toxicity on various organs including endocrine glands. The study aimed to investigate endocrine disorders in patients with Beta-thalassemia major in the Southern Khorasan province, Iran. Materials and Methods In this des...

Pulmonary function tests were performed on 62 transfusion-dependent patients with thalassemia major, ranging in age from 8 to 33 years, and receiving chelation therapy with desferrioxamine or deferiprone. Percent predicted values for FVC, FEV1, and PEF were significantly reduced, whereas FEV1/FVC and maximal expiratory flow at 25% FVC were within normal limits, indicating a restrictive disease....

Background: Thalassemia/haemoglobinopathy is a hereditary disease with increased erythropoiesis and expansion of the bone marrow cavity. Consequently, there is a reduction in trabecular bone tissue resulting in osteopenia/osteoporosis. The present study was performed to determine bone mineral density (BMD) in children and adolescents with major thalassemia and its correlation with serum fer...

background: thalassemia/haemoglobinopathy is a hereditary disease with increased erythropoiesis and expansion of the bone marrow cavity. consequently, there is a reduction in trabecular bone tissue resulting in osteopenia/osteoporosis. the present study was performed to determine bone mineral density (bmd) in children and adolescents with major thalassemia and its correlation with serum ferriti...

-thalassemias are the most common single gene disorders and are potentially amenable to gene therapy. However, retroviral vectors carrying the human -globin cassette have been notoriously unstable. Recently, considerable progress has been made using lentiviral vectors, which stably transmit the -globin expression cassette. Thus far, mouse studies have shown correction of the -thalassemia interm...

beta-thalassemias are the most common single gene disorders and are potentially amenable to gene therapy. However, retroviral vectors carrying the human beta-globin cassette have been notoriously unstable. Recently, considerable progress has been made using lentiviral vectors, which stably transmit the beta-globin expression cassette. Thus far, mouse studies have shown correction of the beta-th...

background: thalassemia is a chronic, inherited blood disorder, which in its most severe form, causes life-threatening anemia. thalassemia patients not only engage with difficulties of blood transfusion and iron chelating therapy but also have some social challenges and health threatening factors. there are some reports on quality of life in thalassemia patients around the world from southeast ...

A preclinical humanized mouse model of thalassemia major or Cooley anemia (CA) was generated by targeted gene replacement of the mouse adult globin genes in embryonic stem cells. The mouse adult and globin genes were replaced with adult human globin genes ( 2 1) and a human fetal to adult hemoglobin (Hb)–switching cassette ( HPFH 0), respectively. Similar to human infants with CA, fully humaniz...