Methods for increasing fetal hemoglobin content in eukaryotic cells

The clinical severity of β-hemoglobinopathies is alleviated by the co-inheritance of genetic mutations causing a sustained fetal γ-globin chain […]

Correction of beta-thalassemia phenotype by genetically engineered

The present invention relates to a genetically modified hematopoietic stem cell (HSC) comprising, in at least one α-globin gene comprised in the […]

Free α-hemoglobin as biomarker for diagnosing and stratifiying

The normal development of red blood cells requires a coordinated synthesis of the hemoglobin (Hb) subunits, the α- and β-globins in the case of […]