Thalassaemia
A Kattamis, JL Kwiatkowski, Y Aydinok - The lancet, 2022 - thelancet.com
Thalassaemia is a diverse group of genetic disorders with a worldwide distribution affecting
globin chain synthesis. The pathogenesis of thalassaemia lies in the unbalanced globin …
globin chain synthesis. The pathogenesis of thalassaemia lies in the unbalanced globin …
Iron metabolism: pathophysiology and pharmacology
K Roemhild, F von Maltzahn, R Weiskirchen… - Trends in …, 2021 - cell.com
Iron is essential in many physiological processes, including DNA metabolism, oxygen
transport, and cellular energy generation. Deregulated iron metabolism, which results in iron …
transport, and cellular energy generation. Deregulated iron metabolism, which results in iron …
[HTML][HTML] CRISPR-Cas9 gene editing for sickle cell disease and β-thalassemia
H Frangoul, D Altshuler, MD Cappellini… - … England Journal of …, 2021 - Mass Medical Soc
Transfusion-dependent β-thalassemia (TDT) and sickle cell disease (SCD) are severe
monogenic diseases with severe and potentially life-threatening manifestations. BCL11A is …
monogenic diseases with severe and potentially life-threatening manifestations. BCL11A is …
Efficacy and safety of luspatercept versus epoetin alfa in erythropoiesis-stimulating agent-naive, transfusion-dependent, lower-risk myelodysplastic syndromes …
Summary Background Erythropoiesis-stimulating agents (ESAs) are the standard-of-care
treatment for anaemia in most patients with lower-risk myelodysplastic syndromes but …
treatment for anaemia in most patients with lower-risk myelodysplastic syndromes but …
Exagamglogene Autotemcel for Transfusion-Dependent β-Thalassemia
F Locatelli, P Lang, D Wall, R Meisel… - … England Journal of …, 2024 - Mass Medical Soc
Background Exagamglogene autotemcel (exa-cel) is a nonviral cell therapy designed to
reactivate fetal hemoglobin synthesis through ex vivo clustered regularly interspaced short …
reactivate fetal hemoglobin synthesis through ex vivo clustered regularly interspaced short …
[HTML][HTML] Long-term outcomes of lentiviral gene therapy for the β-hemoglobinopathies: the HGB-205 trial
E Magrin, M Semeraro, N Hebert, L Joseph… - Nature Medicine, 2022 - nature.com
Sickle cell disease (SCD) and transfusion-dependent β-thalassemia (TDT) are the most
prevalent monogenic disorders worldwide. Trial HGB-205 (NCT02151526) aimed at …
prevalent monogenic disorders worldwide. Trial HGB-205 (NCT02151526) aimed at …
β-Thalassemias
AT Taher, KM Musallam… - New England Journal of …, 2021 - Mass Medical Soc
β-Thalassemias Defective synthesis of the β-globin chain causes recessively inherited
disorders characterized by inadequate hemoglobin production and chronic anemia …
disorders characterized by inadequate hemoglobin production and chronic anemia …
TGF-β: A novel predictor and target for anti-PD-1/PD-L1 therapy
Transforming growth factor-β (TGF-β) signaling regulates multiple physiological processes,
such as cell proliferation, differentiation, immune homeostasis, and wound healing. Besides …
such as cell proliferation, differentiation, immune homeostasis, and wound healing. Besides …
Safety and efficacy of luspatercept for the treatment of anemia in patients with myelofibrosis
AT Gerds, C Harrison, JJ Kiladjian, R Mesa… - Blood …, 2024 - ashpublications.org
Abstract The ACE-536-MF-001 trial enrolled patients with myelofibrosis (n= 95) into 4
cohorts: patients in cohorts 1 and 3A were non–transfusion dependent (NTD) and had …
cohorts: patients in cohorts 1 and 3A were non–transfusion dependent (NTD) and had …
Ineffective erythropoiesis and its treatment
M Cazzola - Blood, The Journal of the American Society of …, 2022 - ashpublications.org
The erythroid marrow and circulating red blood cells (RBCs) are the key components of the
human erythron. Abnormalities of the erythron that are responsible for anemia can be …
human erythron. Abnormalities of the erythron that are responsible for anemia can be …