Hemoglobin subunit beta mRNA (HBB mRNA) (HBB mRNA)

Hemoglobin subunit beta (HBB) mRNA is the messenger RNA transcript that encodes the beta-globin polypeptide, a vital component of the adult hemoglobin (HbA) tetramer responsible for oxygen transport in red blood cells (UniProt: P68871). This molecule is central to the pathophysiology of major genetic hemoglobinopathies, including sickle cell disease and beta-thalassemia, which arise from mutations in the HBB gene that lead to defective or absent beta-globin production (NIH: HBB Gene). In sickle cell disease, a specific point mutation (Glu6Val) results in the production of sickle hemoglobin (HbS), which polymerizes under deoxygenated conditions, causing red blood cells to deform and obstruct microvasculature. Therapeutic strategies targeting HBB mRNA primarily involve gene addition therapies, such as lovotibeglogene autotemcel (Lyfgenia) and betibeglogene autotemcel (Zynteglo), which utilize lentiviral vectors to deliver a functional HBB gene into a patient's hematopoietic stem cells (FDA: Lyfgenia Approval, 2023). These functional genes are then transcribed into healthy HBB mRNA, enabling the synthesis of normal beta-globin and restoring effective oxygen transport to alleviate chronic anemia and vaso-occlusive complications.

Other names

Beta-globin mRNAHBB transcriptHemoglobin beta chain mRNABeta-globin messenger RNA

Lentiviral-mediated gene addition to restore functional HBB mRNA expression and subsequent beta-globin protein synthesis in hematopoietic stem cells.