Sickle cell disease changes adult beta-globin. Instead of two alpha and two healthy beta chains, sickle cell disease patients have two alpha and two mutant beta chains. The resulting hemoglobin is ...

The cells were transduced with a lentiviral vector delivering the gene encoding the anti-sickling beta globin HbAS3 protein. The investigators showed that lentiviral transduction was enhanced by ...

LentiGlobin BB305 Drug Product: LentiGlobin BB305 Drug Product is defined as an autologous CD34+ cell-enriched population that contains HSCs transduced with LentiGlobin BB305 LVV encoding the ...

By birth, γ-globin production is normally switched off, but a significant fraction of RBCs still contain a mixture of fetal and adult hemoglobin. Because the sickle-cell mutation is present only ...

The following is a summary of “Evaluated NSUN3 in reticulocytes from HbH-CS disease that reflects cellular stress in ...

Aruvant’s lead candidate, ARU-1801, consists of autologous cells that are genetically modified with a lentiviral vector that encodes a novel, highly potent anti-sickling γ-globin. ARU-1801 was ...

The causative mutation is the substitution of valine for glutamic acid at the sixth position of the β globin chain (Glu6Val). Sickle cell hemoglobin (HbS) behaves like normal hemoglobin when ...

Beta Thalassemia occurs due to a mutation in the beta globin gene, which is essential for production of normal haemoglobin, the protein inside our red blood cells which carries oxygen from the ...