SRT8-T87Q

SRT8-T87Q (also known as MiNiRoLu) is an optimized lentiviral vector (LVV) designed for the treatment of severe β-hemoglobin disorders, including sickle cell disease (SCD) and transfusion-dependent β-thalassemia. Developed by researchers at Southern Illinois University School of Medicine, the National Institutes of Health (NIH), and the University of Tennessee Health Science Center, the vector is an engineered derivative of the TNS9.3.55 LVV. It features a streamlined genome size of less than 6.9 kb, achieved by repositioning the central polypurine tract (cppt), truncating hypersensitive sites (HS3 and HS4) within the locus control region (LCR), and utilizing a minimal β-globin promoter. The vector encodes the anti-sickling T87Q variant of the β-globin gene. SRT8-T87Q is intended to improve upon existing LVV therapies by offering higher viral titers and enhanced gene transfer efficiency in hematopoietic stem cells (HSCs).

Other names

MiNiRoLu