BCL11a shRNAmiR

BCL11a shRNAmiR is an investigational autologous gene therapy developed by bluebird bio in collaboration with Boston Children's Hospital for the treatment of sickle cell disease and potentially beta-thalassemia. The therapy utilizes a lentiviral vector (specifically the BCH-BB694 vector) to deliver a microRNA-adapted short hairpin RNA (shRNAmiR) into a patient's own CD34+ hematopoietic stem cells ex vivo. This shRNAmiR is designed to achieve erythroid lineage-specific knockdown of BCL11A, a transcription factor that acts as a repressor of fetal hemoglobin (HbF) expression. By targeting BCL11A mRNA through an RNA interference (RNAi) mechanism, the therapy reactivates gamma-globin gene expression and induces the production of fetal hemoglobin, which inhibits the polymerization of sickle hemoglobin (HbS). The treatment process involves harvesting the patient's stem cells, transducing them with the lentiviral vector, and reinfusing the modified cells following myeloablative conditioning.

Other names

microRNA-adapted short hairpin RNA targeting BCL11A