ERAD-focused research article presenting a platform of small-molecule–responsive gene switches for rapidly controllable protein secretion and transgene regulation. Open to learn how this approach could enable on-demand control of therapeutic activities in vivo.
Key Takeaways
- Conditional degron tags enabled oral small-molecule control of native protein secretion in vivo
- Systems were compatible with AAV delivery enabling long-term remote-controlled transgene expression in male mice
- Applications demonstrated include controllable gene therapies for cardiovascular disease and chronic pain