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The Scope for Thalassemia Gene Therapy by Disruption of Aberrant Regulatory Elements
Open AccessReview

Erythropoiesis In Vitro—A Research and Therapeutic Tool in Thalassemia

The Hematology Department, Hadassah—Hebrew University Medical Center, Jerusalem 91120, Israel
J. Clin. Med. 2019, 8(12), 2124; https://doi.org/10.3390/jcm8122124
Received: 2 November 2019 / Revised: 26 November 2019 / Accepted: 29 November 2019 / Published: 2 December 2019
(This article belongs to the Special Issue New Trends in Personalized Therapy of Thalassemia)
Thalassemia (thal) is a hereditary chronic hemolytic anemia due to a partial or complete deficiency in the production of globin chains, in most cases, α or β, which compose, together with the iron-containing porphyrins (hemes), the hemoglobin molecules in red blood cells (RBC). The major clinical symptom of β-thal is severe chronic anemia—a decrease in RBC number and their hemoglobin content. In spite of the improvement in therapy, thal still severely affects the quality of life of the patients and their families and imposes a substantial financial burden on the community. These considerations position β-thal, among other hemoglobinopathies, as a major health and social problem that deserves increased efforts in research and its clinical application. These efforts are based on clinical studies, experiments in animal models and the use of erythroid cells grown in culture. The latter include immortal cell lines and cultures initiated by erythroid progenitor and stem cells derived from the blood and RBC producing (erythropoietic) sites of normal and thal donors, embryonic stem cells, and recently, "induced pluripotent stem cells" generated by manipulation of differentiated somatic cells. The present review summarizes the use of erythroid cultures, their technological aspects and their contribution to the research and its clinical application in thal. The former includes deciphering of the normal and pathological biology of the erythroid cell development, and the latter—their role in developing innovative therapeutics—drugs and methods of gene therapy, as well as providing an alternative source of RBC that may complement or substitute blood transfusions.
Keywords: thalassemia; erythroid cells; cultures; hemoglobin thalassemia; erythroid cells; cultures; hemoglobin
MDPI and ACS Style

Fibach, E. Erythropoiesis In Vitro—A Research and Therapeutic Tool in Thalassemia. J. Clin. Med. 2019, 8, 2124.

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