Catherine Skari – Page 38

Jan- 2021 -

21 January

CRISPR-Cas9 Gene Editing for Sickle Cell Disease and β-Thalassemia

Transfusion-dependent β-thalassemia (TDT) and sickle cell disease (SCD) are severe monogenic diseases with severe and potentially life-threatening manifestations. BCL11A is…

12 January

Scientific Literature

The Use of Luspatercept for Thalassemia in Adults

Luspatercept is an activin receptor ligand trap that has been shown to enhance late-stage erythropoiesis in animal models of β-thalassemia.…

8 January

Scientific Literature

The Prevention of Thalassemia Revisited: A Historical and Ethical Perspective by the Thalassemia International Federation

Hemoglobinopathies are the most common monogenic disorders in humans; among them, thalassemia constitutes a serious medical and public health problem…

5 January

Scientific Literature

Nutrition in Thalassemia: A Systematic Review of Deficiency, Relations to Morbidity, and Supplementation Recommendations

Reports of nutritional deficiencies in patients with thalassemia (Thal) are common. Despite its importance, however, nutritionally focused research in Thal…

Sep- 2020 -

30 September

Scientific Literature

Fair Pricing of Innovative Medicines: An EHA Position Paper

High prices keep innovative medicines out of reach for many patients across Europe, resulting in growing inequalities in accessibility and…

11 September

Scientific Literature

Gene Therapy of the Hemoglobinopathies

Sickle cell disease and the ß-thalassemias are caused by mutations of the ß-globin gene and represent the most frequent single…

Aug- 2020 -

27 August

Scientific Literature

Challenging the Erythropoiesis Paradigm in β-Thalassemia

β-thalassemia is a genetic disorder due to defective β-globin synthesis that results in severe anemia. A hallmark of the disease…

15 August

Scientific Literature

Effects of A-Tocopherol on Hemolysis and Oxidative Stress Markers on Red Blood Cells in β-Thalassemia Major

The accumulation of unpaired α-globin chains in patients with β-thalassemia major may clinically create ineffective erythropoiesis, hemolysis, and chronic anemia.…

12 August

Scientific Literature

Recommendations for Pregnancy in Rare Inherited Anemias

Rare inherited anemias are a subset of anemias caused by a genetic defect along one of the several stages of…

May- 2020 -

27 May

Scientific Literature

Update in Laboratory Diagnosis of Thalassemia

Alpha- and β-thalassemias and abnormal hemoglobin (Hb) are common in tropical countries. These abnormal globin genes in different combinations lead…