Article

Expression of γ-globin genes in β-thalassemia patients treated with sirolimus: results from a pilot clinical trial (Sirthalaclin)

2021-12-21

Abstract excerpt

<h4>Introduction</h4> The β-thalassemias are due to autosomal mutations of the β-globin gene, inducing absence or low-level synthesis of β-globin in erythroid cells. It is widely accepted that high production of fetal hemoglobin (HbF) is beneficial for β-thalassemia patients. Sirolimus, also known as rapamycin, is a lipophilic macrolide isolated from a strain of Streptomyces hygroscopicus found to be a strong HbF...

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Literature Corpus work: afb15186-7ac4-5a39-810e-543ae4943f35
DOI: 10.1101/2021.12.20.21267830

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