Improvement of care for thalassemia during previous decades focussed on the development of new diagnostic and treatment modalities concerning secondary hemochromatosis resulting from symptomatic treatment with regular transfusion. In addition, hematopoetic stem transplantation as the so far only curative approach had been developed and constantly improved. New approaches for the treatment of thalassemias already entering clinical practice include e.g. additive gene therapy and medical treatment with new drugs like luspatercept. For sickle cell disease, a variety of treatment approaches either to correct the genetic change by gene therapy, to affect the underlying mechanism of sickling e.g. by R-state- stabilising agents, or later the pathop...
AbstractBeta-thalassemia is a group of frequent genetic disorders resulting in the synthesis of litt...
Thalassemias (α, β, γ, δ, δβ, and εγδβ) are the most common genetic disorders worldwide and constitu...
Inherited hemoglobin disorders, including beta-thalassemia (BT) and sickle-cell disease (SCD), are t...
Improvement of care for thalassemia during previous decades focussed on the development of new diagn...
β-thalassemias and sickle cell anemia (SCA) are the most common monogenic diseases worldwide for whi...
Hemoglobinopathies, including thalassemias and sickle cell disease, are the most common monogenic di...
High-level production of -globin, -globin, or therapeutic mutant globins in the RBC lineage by hemat...
Despite an increased understanding of the pathophysiology of sickle cell disease (SCD), there remain...
Despite improvements in the management of thalassemia major and sickle cell disease, treatment compl...
β-thalassemias are heterogeneous hereditary anemias characterized by a reduced output of β-globin ch...
Thalassemia major and sickle cell disease are the two most widely disseminated hereditary hemoglobin...
International audienceBêta-globin gene transfer has been used as a paradigm for hematopoietic stem c...
Sickle cell disease and its variants constitute the most common inherited blood disorders affecting ...
Beta-thalassemia is a group of frequent genetic disorders resulting in the synthesis of little or no...
The main characteristic of the pathophysiology of β-thalassemia is reduced β-globin chain production...
AbstractBeta-thalassemia is a group of frequent genetic disorders resulting in the synthesis of litt...
Thalassemias (α, β, γ, δ, δβ, and εγδβ) are the most common genetic disorders worldwide and constitu...
Inherited hemoglobin disorders, including beta-thalassemia (BT) and sickle-cell disease (SCD), are t...
Improvement of care for thalassemia during previous decades focussed on the development of new diagn...
β-thalassemias and sickle cell anemia (SCA) are the most common monogenic diseases worldwide for whi...
Hemoglobinopathies, including thalassemias and sickle cell disease, are the most common monogenic di...
High-level production of -globin, -globin, or therapeutic mutant globins in the RBC lineage by hemat...
Despite an increased understanding of the pathophysiology of sickle cell disease (SCD), there remain...
Despite improvements in the management of thalassemia major and sickle cell disease, treatment compl...
β-thalassemias are heterogeneous hereditary anemias characterized by a reduced output of β-globin ch...
Thalassemia major and sickle cell disease are the two most widely disseminated hereditary hemoglobin...
International audienceBêta-globin gene transfer has been used as a paradigm for hematopoietic stem c...
Sickle cell disease and its variants constitute the most common inherited blood disorders affecting ...
Beta-thalassemia is a group of frequent genetic disorders resulting in the synthesis of little or no...
The main characteristic of the pathophysiology of β-thalassemia is reduced β-globin chain production...
AbstractBeta-thalassemia is a group of frequent genetic disorders resulting in the synthesis of litt...
Thalassemias (α, β, γ, δ, δβ, and εγδβ) are the most common genetic disorders worldwide and constitu...
Inherited hemoglobin disorders, including beta-thalassemia (BT) and sickle-cell disease (SCD), are t...