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A modified cyclosporine enhances lentivector transduction ex vivo and in vivo by degrading IFITM3

Cellular antiviral defenses limit lentivector gene delivery. The authors describe lentivector transduction enhancement via inhibition of antiviral IFITM3 by novel inhibitor BG147. BG147 has activity ex vivo in human stem cells and in vivo in mouse subretinal cells, driving IFITM3 degradation via lysosomes. BG147 can expand lentivector utility and reduce treatment costs.
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