Protein replacement is a well established therapeutic strategy although delivering to the brain remains a challenge. An example includes providing insulin for diabetics. Since proteins degrade over time this therapeutic would need to be provided on an ongoing basis.
What it is
Rather than intervene at the DNA or RNA level, this strategy introduces healthy MECP2 protein to compensate for the mutated one. This strategy is not a one and done, but would need to be administered on an ongoing basis.
Why it matters
Protein replacement is a well established modality which 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.
The protein replacement program we are funding is leveraging a novel mechanism to traffic the healthy MECP2 protein into the brain with unprecedented efficiency.
A cure for Rett.
You want it.
We want it.
Great achievements happen when people stand up and say I’m in. I will be part of the solution!
Rett may be a rare disorder but together we are powerful.