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

Cristina ZuccatoLucia Carmela CosenzaMatteo ZurloJessica GasparelloChiara PapiElisabetta D'AversaGiulia BreveglieriIlaria LamprontiAlessia FinottiMonica BorgattiChiara ScapoliAlice StievanoMonica FortiniEric RamazzottiNicola MarchettiMarco ProsdocimiMaria Rita GamberiniRoberto Gambari
Published in: Therapeutic advances in hematology (2022)
The data obtained indicate that low doses of sirolimus modify hematopoiesis and induce increased expression of γ-globin genes in a subset of patients with β-thalassemia. Further clinical trials are warranted, possibly including testing of the drug in patients with less severe forms of the disease and exploring combination therapies.
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