To better understand human health and disease, researchers create a wide variety of mouse
models that carry human DNA. With recent advances in genome engineering, the targeted …

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 …

Disorders involving β-globin gene mutations, primarily β-thalassemia and sickle cell
disease, represent a major target for hematopoietic stem/progenitor cell (HSPC) gene …

Minihepcidins are hepcidin agonists that have been previously shown to reverse iron
overload and improve erythropoiesis in mice affected by non-transfusion-dependent …

In utero gene therapy (IUGT) to the fetal hematopoietic compartment could be used to treat
congenital blood disorders such as β-thalassemia. A humanised mouse model of β …

Abstract β-thalassemia (βT) is a genetic blood disorder causing profound and life
threatening anemia. Current clinical management of βT is a lifelong dependence on regular …

Advanced therapy medicinal products (ATMPs) are medicines for human use based on
genes, cells or tissue engineering. After clear successes in adults, the nascent technology …

Introduction: Beta-thalassemias are caused by more than 200 mutations of the β-globin
gene, leading to low or absent production of adult hemoglobin. Achievements have been …

GFP is frequently used as a marker for tracking donor cells adoptively transplanted into
recipient animals. The human ubiquitin C promoter (UBC)–driven-GFP transgenic mouse is …

Elevated levels of fetal γ-globin can cure disorders caused by mutations in the adult β-globin
gene. This clinical finding has motivated studies to improve our understanding of …