Gene therapy for hemoglobinopathies
MR Lidonnici, S Scaramuzza, G Ferrari - Human Gene Therapy, 2023 - liebertpub.com
β-Thalassemia and sickle cell disease are autosomal recessive disorders of red blood cells
due to mutations in the adult β-globin gene, with a worldwide diffusion. The severe forms of …
due to mutations in the adult β-globin gene, with a worldwide diffusion. The severe forms of …
[HTML][HTML] Treatment of sickle cell disease-options and perspective
L Abdel-Hadi, YV Carmenate… - American Journal of …, 2023 - ncbi.nlm.nih.gov
Abstract Sickle Cell Disease (SCD) is one of the most inherited hematologic diseases
affecting humans. Clinically, there is a progressive multiorgan failure and increased mortality …
affecting humans. Clinically, there is a progressive multiorgan failure and increased mortality …
[HTML][HTML] Genetic regulation of fetal hemoglobin across global populations
Human genetic variation has enabled the identification of several key regulators of fetal-to-
adult hemoglobin switching, including BCL11A, resulting in therapeutic advances. However …
adult hemoglobin switching, including BCL11A, resulting in therapeutic advances. However …
New Insights in the Treatment of Sickle Cell Disease in Childhood
S Bittmann, E Luchter… - Asian Journal of …, 2024 - archive.sdpublishers.com
Sickle cell disease encompasses diseases that are pathophysiologically caused by
hemoglobin S. The HbS component of total hemoglobin in SCD is normally over 50%. HbS …
hemoglobin S. The HbS component of total hemoglobin in SCD is normally over 50%. HbS …
Antioxidative Potential of Foetal Haemoglobin in Sickle Cell Disease
YI Dogonzo, CC Onyeabor, CM Oru… - … of Research and …, 2023 - article.researchpromo.com
Background: Oxidative stress is a clinical condition in sickle cell disease (SCD) that results
from increased production of reactive oxygen species (ROS). High Foetal haemoglobin …
from increased production of reactive oxygen species (ROS). High Foetal haemoglobin …