What it is
Protein replacement introduces healthy MECP2 protein to compensate for the mutated one. An example of a replacement strategy includes providing insulin for diabetics.This strategy is not a one-and-done strategy but would need to be administered on an ongoing basis.
Why it's part of our plan
Protein replacement is a well established modality that can currently treat cancer, diabetes, autoimmune disorders, blood diseases, and many other disorders. Protein replacement for Rett would make it possible to titrate the dose to the individual, allowing just the right amount to improve their symptoms. Since proteins degrade over time, this therapeutic would need to be provided on an ongoing basis.
The biggest challenge with this strategy is delivering sufficient amounts of MECP2 protein to the entire brain.
RSRT is funding the work of Dr. Alanna Schepartz of UC Berkeley, whose program is leveraging a novel mechanism to traffic the healthy MECP2 protein into the brain with unprecedented efficiency.