Vector rearrangement boosts globin-delivering gene therapies

NIH vector rearrangement boosts production, transduction efficiencies of lentiviral β globin delivery

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

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