Recent trends in the gene therapy of β-thalassemia
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 …
mutations of the adult β-globin gene. Together with sickle cell anemia, thalassemia …
Do genetic polymorphisms affect fetal hemoglobin (HbF) levels in patients with sickle cell anemia treated with hydroxyurea? A systematic review and pathway analysis
RR Sales, BL Nogueira, JAG Tosatti… - Frontiers in …, 2022 - frontiersin.org
Hydroxyurea has long been used for the treatment of sickle cell anemia (SCA), and its
clinical effectiveness is related to the induction of fetal hemoglobin (HbF), a major modifier of …
clinical effectiveness is related to the induction of fetal hemoglobin (HbF), a major modifier of …
[HTML][HTML] Frequency of hospitalizations for pain and association with altered brain network connectivity in sickle cell disease
Sickle cell disease (SCD) is a hemoglobinopathy that affects more than 100,000 individuals
in the United States. The disease is characterized by the presence of sickle hemoglobin and …
in the United States. The disease is characterized by the presence of sickle hemoglobin and …
Three fingers on the switch: Krüppel-like factor 1 regulation of: γ-globin: to: β-globin: gene switching
MR Tallack, AC Perkins - Current opinion in hematology, 2013 - journals.lww.com
KLF1 regulates the γ-globin to β-globin genetic switch by many mechanisms. Firstly, it
facilitates formation of an active chromatin hub (ACH) at the β-globin gene cluster …
facilitates formation of an active chromatin hub (ACH) at the β-globin gene cluster …
Advances in technologies for screening and diagnosis of hemoglobinopathies
J Traeger-Synodinos, CL Harteveld - Biomarkers in Medicine, 2014 - Taylor & Francis
Hemoglobinopathies constitute the most common monogenic disorders worldwide, caused
by mutations in the globin genes that synthesize the globin chains of hemoglobin. Synthesis …
by mutations in the globin genes that synthesize the globin chains of hemoglobin. Synthesis …
Hydroxyurea in the management of sickle cell disease: pharmacogenomics and enzymatic metabolism
SCMA Yahouédéhou, EV Adorno… - The …, 2018 - nature.com
Hydroxyurea (HU) was approved to be used in the treatment of sickle cell disease (SCD)
because of its anti-sickling potential. However, there is variability in HU response among …
because of its anti-sickling potential. However, there is variability in HU response among …
Genome-based therapeutic interventions for β-type hemoglobinopathies
For decades, various strategies have been proposed to solve the enigma of
hemoglobinopathies, especially severe cases. However, most of them seem to be lagging in …
hemoglobinopathies, especially severe cases. However, most of them seem to be lagging in …
Pharmacological and molecular approaches for the treatment of β‐hemoglobin disorders
N Lohani, N Bhargava, A Munshi… - Journal of cellular …, 2018 - Wiley Online Library
β‐hemoglobin disorders, such as β‐thalassemia and sickle cell anemia are among the most
prevalent inherited genetic disorders worldwide. These disorders are caused by mutations …
prevalent inherited genetic disorders worldwide. These disorders are caused by mutations …
Distinct miRNA signatures and networks discern fetal from adult erythroid differentiation and primary from immortalized erythroid cells
MicroRNAs (miRNAs) are small non-coding RNAs crucial for post-transcriptional and
translational regulation of cellular and developmental pathways. The study of miRNAs in …
translational regulation of cellular and developmental pathways. The study of miRNAs in …
Preconception carrier screening and prenatal diagnosis in thalassemia and hemoglobinopathies: challenges and future perspectives
J Traeger-Synodinos, CL Harteveld - Expert review of molecular …, 2017 - Taylor & Francis
Introduction: Hemoglobinopathies constitute the most common severe monogenic disorders
worldwide, with an increasing global burden each year. The benefit of applying programmes …
worldwide, with an increasing global burden each year. The benefit of applying programmes …