A Destabilizing Domain Allows for Fast Noninvasive Conditional Control of Protein Abundance in the Mouse Eye – Implications for Ocular Gene Therapy

摘要

PurposeTemporal and reversible control of protein expression in vivo is a central goal for many gene therapies, especially for strategies involving proteins that are detrimental to physiology if constitutively expressed. Accordingly, we explored whether protein abundance in the mouse retina could be effectively controlled using a destabilizing Escherichia coli dihydrofolate reductase (DHFR) domain whose stability is dependent on the small molecule, trimethoprim (TMP).