The Long Scientific Journey of Sirolimus (Rapamycin): From the Soil of Easter Island (Rapa Nui) to Applied Research and Clinical Trials on β-Thalassemia and Other Hemoglobinopathies

The Long Scientific Journey of Sirolimus (Rapamycin): From the Soil of Easter Island (Rapa Nui) to Applied Research and Clinical Trials on β-Thalassemia and Other Hemoglobinopathies

In this review article, we present the fascinating story of rapamycin (sirolimus), a drug able to induce γ-globin gene expression and increased production of fetal hemoglobin (HbF) in erythroid cells, including primary erythroid precursor cells (ErPCs) isolated from β-thalassemia patients. For this...

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Bibliographic Details
Main Authors: Roberto Gambari, Cristina Zuccato, Lucia Carmela Cosenza, Matteo Zurlo, Jessica Gasparello, Alessia Finotti, Maria Rita Gamberini, Marco Prosdocimi
Format: Article
Language:English
Published: MDPI AG 2023-09-01
Series:Biology
Subjects:
β-thalassemia
γ-globin genes
rapamycin (sirolimus)
fetal hemoglobin
clinical trials
Online Access:https://www.mdpi.com/2079-7737/12/9/1202

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