[HTML][HTML] Molecular genetics of β-thalassemia: A narrative review

TH Jaing, TY Chang, SH Chen, CW Lin, YC Wen… - Medicine, 2021 - journals.lww.com
Abstract β-thalassemia is a hereditary hematological disease caused by over 350 mutations
in the β-globin gene (HBB). Identifying the genetic variants affecting fetal hemoglobin (HbF) …

Beyond hydroxyurea: new and old drugs in the pipeline for sickle cell disease

MJ Telen - Blood, The Journal of the American Society of …, 2016 - ashpublications.org
Abstract Despite Food and Drug Administration (FDA) approval of hydroxyurea to reduce the
frequency of vaso-occlusive episodes, sickle cell disease (SCD) has continued to be treated …

Inflammation in sickle cell disease

N Conran, JD Belcher - Clinical hemorheology and …, 2018 - journals.sagepub.com
The primary β-globin gene mutation that causes sickle cell disease (SCD) has significant
pathophysiological consequences that result in hemolytic events and the induction of the …

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 …

Allogeneic hematopoietic stem cell transplantation to cure sickle cell disease: a review

N Bhalla, A Bhargav, SK Yadav, AK Singh - Frontiers in Medicine, 2023 - frontiersin.org
Sickle cell disease (SCD) had first been mentioned in the literature a century ago.
Advancement in the molecular basis of the pathophysiology of the disease opens the door …

Sodium butyrate mitigates type 2 diabetes by inhibiting PERK-CHOP pathway of endoplasmic reticulum stress

Y Hu, J Liu, Y Yuan, J Chen, S Cheng, H Wang… - Environmental …, 2018 - Elsevier
Sodium butyrate (NaB), a histone deacetylase (HDAC) inhibitors, has been reported to
attenuate hyperglycemia in rats. Our objective was to explore the effect and underlying …

Thalassemia an update: molecular basis, clinical features and treatment

K Tari, P Valizadeh Ardalan… - International journal of …, 2018 - ijbmph.com
Thalassemia are a group of inherited blood disorders caused by the decrease or absence of
beta-globin chain synthesis will be determined with decrease in erythrocyte hemoglobin …

Pathophysiology and recent therapeutic insights of sickle cell disease

F Shah, M Dwivedi - Annals of Hematology, 2020 - Springer
Sickle cell disease (SCD) is an autosomal recessive blood disorder which occurs due to
point mutation in the β-globin chain of hemoglobin. Since the past decades, various …

Hydroxyurea down-regulates BCL11A, KLF-1 and MYB through miRNA-mediated actions to induce γ-globin expression: implications for new therapeutic …

GD Pule, S Mowla, N Novitzky, A Wonkam - Clinical and translational …, 2016 - Springer
Background The major therapeutic benefit of hydroxyurea, the only FDA-approved
pharmacologic treatment for sickle cell disease (SCD), is directly related to fetal hemoglobin …

MicroRNA expression in β-thalassemia and sickle cell disease: a role in the induction of fetal hemoglobin

N Saki, S Abroun, M Soleimani… - Cell Journal …, 2016 - pmc.ncbi.nlm.nih.gov
Today the regulatory role of microRNAs (miRs) is well characterized in many diverse cel-
lular processes. MiR-based regulation is categorized under epigenetic regulatory mecha …