HybHBB-tEPOR gene therapy

HybHBB-tEPOR gene therapy is an experimental ex vivo gene editing platform developed by researchers at Stanford University for the treatment of β-hemoglobinopathies, including sickle cell disease and β-thalassemia. The therapy utilizes a CRISPR-Cas9 and AAV6-mediated approach to replace the endogenous HBB gene via homology-directed repair (HDR). The construct features a hybrid β-globin transgene (HybHBB), consisting of HBB exons 1–3 and heterologous γ-globin introns, linked to a truncated erythropoietin receptor (tEPOR). The tEPOR component is designed to provide a selective expansion advantage to edited erythroid cells, allowing for high levels of functional correction even at low vector doses, which minimizes p53-mediated cellular stress and preserves hematopoietic stem cell fitness.

Other names

HybHBB-tEPOR construct