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Transfusion independence and HMGA2 activation after gene therapy of human β-thalassaemia
The β-haemoglobinopathies are the most prevalent inherited disorders worldwide. Gene therapy of β-thalassaemia is particularly challenging given the requirement for massive haemoglobin production inExpand
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Impact of early transcranial Doppler screening and intensive therapy on cerebral vasculopathy outcome in a newborn sickle cell anemia cohort.
Transcranial Doppler (TCD) is used to detect children with sickle cell anemia (SCA) who are at risk for stroke, and transfusion programs significantly reduce stroke risk in patients with abnormalExpand
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The natural history of asymptomatic osteonecrosis of the femoral head in adults with sickle cell disease.
BACKGROUND Osteonecrosis of the femoral head is a frequent complication in adult patients with sickle cell disease. However, little is known about the natural history of asymptomatic lesions. Expand
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Endothelial dysfunction in patients with sickle cell disease is related to selective impairment of shear stress-mediated vasodilation.
Interactions between the endothelium and erythrocytes may contribute to the vascular complications of sickle cell disease (SCD). Endothelium-derived nitric oxide (NO) plays a major role in theExpand
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Influence of sickle cell disease and treatment with hydroxyurea on sperm parameters and fertility of human males
The use of hydroxyurea has considerably modified the prognosis of sickle cell disease and many more patients now reach reproductive age. This study shows alterations of semen parameters due to sickleExpand
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Sickle-Cell Disease in Brazzaville, Congo: Genetical, Hematological, Biochemical and Clinical Aspects
Clinical, hematological and molecular features of 116 unrelated sickle-cell anemia patients from Brazzaville were investigated. The mean age of the patients was 9.4 ± 5.3 years. 232 βs-chromosomesExpand
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Improvements in haemolysis and indicators of erythrocyte survival do not correlate with acute vaso‐occlusive crises in patients with sickle cell disease: a phase III randomized, placebo‐controlled,
Red blood cell (RBC) hydration is regulated in part by the Ca2+‐activated K+ efflux (Gardos) channel. Senicapoc selectively blocks potassium efflux through the Gardos channel, reducing RBCExpand
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Pulmonary hypertension and cor pulmonale during severe acute chest syndrome in sickle cell disease.
RATIONALE Steady-state mild pulmonary hypertension is a risk factor for death in adults with sickle cell disease. Acute pulmonary hypertension has been reported during exercise and vasoocclusive painExpand
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Pulmonary function in sickle cell disease with or without acute chest syndrome.
Recurrent acute chest syndrome (ACS) has been suggested as a risk factor for chronic lung dysfunction in sickle cell disease. To investigate this hypothesis, lung function tests were performed in 49Expand
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