A drug-tunable gene therapy for broad-spectrum protection against retinal degeneration
C. P. Santiago, C. J. Keuthan, S. Boye, S. Boye, A. A. Imam, 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.
bioRxiv · Mar 28, 2018Bioengineering