Recent trends in the gene therapy of β-thalassemia

A Finotti, L Breda, CW Lederer, N Bianchi… - Journal of blood …, 2015 - Taylor & Francis
The β-thalassemias are a group of hereditary hematological diseases caused by over 300
mutations of the adult β-globin gene. Together with sickle cell anemia, thalassemia …

[HTML][HTML] Current and future alternative therapies for beta-thalassemia major

E de Dreuzy, K Bhukhai, P Leboulch, E Payen - Biomedical journal, 2016 - Elsevier
Beta-thalassemia is a group of frequent genetic disorders resulting in the synthesis of little or
no β-globin chains. Novel approaches are being developed to correct the resulting α/β …

FOXO3‐mTOR metabolic cooperation in the regulation of erythroid cell maturation and homeostasis

X Zhang, G Campreciós, P Rimmelé… - American journal of …, 2014 - Wiley Online Library
Ineffective erythropoiesis is observed in many erythroid disorders including β‐thalassemia
and anemia of chronic disease in which increased production of erythroblasts that fail to …

Advances in understanding the mechanisms of erythropoiesis in homeostasis and disease

R Liang, S Ghaffari - British journal of haematology, 2016 - Wiley Online Library
Anaemia or decreased blood haemoglobin is the most common blood disorder often
characterized by reduced red blood cell (RBC) numbers. RBC s are produced from …

A systems approach identifies essential FOXO3 functions at key steps of terminal erythropoiesis

R Liang, G Campreciós, Y Kou, K McGrath… - PLoS …, 2015 - journals.plos.org
Circulating red blood cells (RBCs) are essential for tissue oxygenation and homeostasis.
Defective terminal erythropoiesis contributes to decreased generation of RBCs in many …

Standardized microfluidic assessment of red blood cell–mediated microcapillary occlusion: Association with clinical phenotype and hydroxyurea responsiveness in …

Y Man, E Kucukal, R An, A Bode, JA Little… - …, 2021 - Wiley Online Library
Objectives We present a standardized in vitro microfluidic assay and Occlusion Index (OI) for
the assessment of red blood cell (RBC)–mediated microcapillary occlusion and its clinical …

A comprehensive review of hydroxyurea for β-haemoglobinopathies: the role revisited during COVID-19 pandemic

N Yasara, A Premawardhena, S Mettananda - Orphanet Journal of Rare …, 2021 - Springer
Background Hydroxyurea is one of the earliest drugs that showed promise in the
management of haemoglobinopathies that include β-thalassaemia and sickle cell disease …

Elevated CDKN1A (P21) mediates β-thalassemia erythroid apoptosis, but its loss does not improve β-thalassemic erythropoiesis

R Liang, M Lin, V Menon, J Qiu, A Menon… - Blood …, 2023 - ashpublications.org
Abstract β-thalassemias are common hemoglobinopathies due to mutations in the β-globin
gene that lead to hemolytic anemias. Premature death of β-thalassemic erythroid precursors …

Transcription factors FOXO in the regulation of homeostatic hematopoiesis

V Menon, S Ghaffari - Current opinion in hematology, 2018 - journals.lww.com
FOXO are safeguards of homeostatic hematopoiesis. FOXO networks and their regulators
and coactivators in HSPC are greatly complex and less well described. Identifications and …

Repurposing of hydroxyurea against COVID-19: a promising immunomodulatory role

M Ben Moftah, A Eswayah - ASSAY and Drug Development …, 2022 - liebertpub.com
Cytokine release syndrome, a prominent mechanism of morbidity and mortality in patients
with coronavirus disease 2019 (COVID-19), can cause multiple bodily reactions, including …