Article
Efficacy of Rapamycin as Inducer of Hb F in Primary Erythroid Cultures from Sickle Cell Disease and β-Thalassemia Patients.
Hemoglobin - 1 Jan 2015
Pecoraro Alice, Troia Antonio, Calzolari Roberta, Scazzone Concetta, Rigano Paolo, Martorana Adriana, Sacco Massimiliano, Maggio Aurelio, Di Marzo Rosalba
Abstract excerpt
Phenotypic improvement of hemoglobinopathies such as sickle cell disease and β-thalassemia (β-thal) has been shown in patients with high levels of Hb F. Among the drugs proposed to increase Hb F production, hydroxyurea (HU) is currently the only one proven to improve the clinical course of these diseases. However, Hb F increase and patient's response are highly variable, indicating that new pharmacological agents...
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