Vector rearrangement boosts globin-delivering gene therapies

Through vector genome reorganization, NIH researchers have amplified the manufacturing output and β globin delivery for a lentiviral gene therapy for sickle cell disease and β thalassemia.

According to Naoya Uchida, gene therapies delivering β globin (HBB) have been "reverse-oriented" -- arranged such that the HBB construct and vector backbone read in opposite directions -- to prevent splicing from eliminating an enhancer-containing intron required for high HBB expression...