BioengineeringbioRxiv
A drug-tunable gene therapy for broad-spectrum protection against retinal degeneration
C. P. Santiago, C. J. Keuthan, S. Boye…·J. D. Ash
This study develops a drug-tunable gene therapy vector, RPF2, that protects photoreceptors and prevents blindness in mice by controlling transgene expression with trimethoprim.
DOI 10.1016/j.ymthe.2018.07.016