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Human βA-T87Q-globin (βA-T87Q-globin)
- Target
- βA-T87Q-globin
- Molecular classification
- Globin, Hemoglobin subunit
01
Overview
The human βA-T87Q-globin gene is a modified version of the adult β-globin gene (HBB) with a threonine to glutamine substitution at position 87. This modification confers anti-sickling properties, making it a therapeutic target for hemoglobinopathies such as sickle cell disease and transfusion-dependent β-thalassemia. It functions by disrupting HbS polymerization and restoring/augmenting normal hemoglobin function.
- Other names
- HbAT87Qβ-globin T87QAnti-sickling β-globin
02
Mechanism of action
Inhibits HbS polymerization, restoring effective oxygen transport by producing functional hemoglobin tetramers.
03
Biological functions
Oxygen transportHemoglobin formationInhibition of HbS polymerization
04
Disease associations
Sickle cell diseaseTransfusion-dependent β-thalassemia
05
Safety considerations
Insertional mutagenesis (lentiviral vector)Off-target effectsLong-term durability of gene expressionPotential for clonal dominance
06
Interacting drugs
Betibeglogene autotemcel (ZYNTEGLO)
07
Biomarkers
HbAT87Q expression level in red blood cellsTotal hemoglobin levelsPercentage of red blood cells expressing HbAT87Q
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