shRNA-BCL11a-F36VMpl lentiviral vector

shRNA-BCL11a-F36VMpl lentiviral vector is an investigational ex vivo gene therapy designed for the treatment of beta-hemoglobinopathies, including beta-thalassemia and sickle cell disease. Developed by researchers at the Aristotle University of Thessaloniki, Democritus University of Thrace, and George Papanikolaou General Hospital of Thessaloniki, the lentiviral vector utilizes a short-hairpin RNA (shRNA) to target and silence B-cell lymphoma/leukemia 11A (BCL11A), a key transcriptional repressor of fetal hemoglobin (HbF). This silencing leads to the reactivation of gamma-globin and the production of HbF. To overcome the limitations of low transduction efficiency in hematopoietic stem cells (HSCs), the vector incorporates a selective expansion cassette encoding F36VMpl, a mutant thrombopoietin receptor fusion protein. In the presence of a chemical inducer of dimerization (CID) such as AP20187, F36VMpl triggers a growth signal that selectively expands the transduced cell population both in vitro and in vivo.

Other names

shRNA-BCL11A-F36VMplshRNA-BCL-11A-F36VMplshRNA-BCL 11A-F36VMplshRNA-BCL11A-F36VMpl lentiviral vectorshRNA-BCL-11A-F36VMpl lentiviral vectorshRNA-BCL 11A-F36VMpl lentiviral vector