The USFDA had approved betibeglogene autotemcel, the first gene-therapy-based treatment for beta-thalassemia patients (adults and children) who require RBC transfusions. Betibeglogene autotemcel is a lentiviral-vector-based gene therapy that inserts a functional β-globin gene into a patient’s hematopoietic stem cells. It enables them to produce normal to near-normal amounts of total hemoglobin and eliminates the need for frequent RBC transfusions.
S tudy overview Do you think gene therapy has the potential to revolutionize beta-thalassemia treatment? Let us know by commenting below.
