Recent trends in treatment of thalassemia

Blood Cells, Molecules & Diseases
Amal El-Beshlawy, Mona El-Ghamrawy

Abstract

Thalassemia is a common inherited monogenic disease. It is characterized by chronic hemolysis, ineffective erythropoiesis (IE) and iron overload. Despite advances in transfusion practices and chelation therapy, still many limitations in delivering these standard therapies exist. Challenges of currently available standard care and advances in understanding the underlying pathophysiological mechanisms in thalassemia stimulated research towards development of novel therapeutic targets. Agents reducing IE as Jak 2 inhibitors and Activin II receptor traps are promising and are currently in clinical trials. Other approaches targeting iron dysregulation as mini-hepcidins, exogenous transferrin and erythroferrone inhibitors are in preclinical studies. Gene therapy, a rapidly evolving field, has exhibited remarkable progress in recent years. Studies have focused on β or γ-globin addition, over expression of endogenous γ-globin-activating transcription factors, silencing of γ-globin repressors and genome editing of β-globin mutations or γ-globin repressors. In this article we provide an overview of emerging recent trends in treatment of thalassemia targeting IE, iron dysregulation and novel curative treatments as gene therapy and gene ed...Continue Reading

Citations

Apr 7, 2020·Expert Opinion on Emerging Drugs·Rayan Bou-FakhredinAli T Taher
Dec 14, 2019·Blood Cells, Molecules & Diseases·Boris E ShmuklerSeth L Alper
May 18, 2021·Molecular Therapy. Methods & Clinical Development·Lucia Carmela CosenzaAlessia Finotti

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