My newborn was diagnosed with Sturge–Weber syndrome and mRNA sounds like a potential solution.
The issue is it's rare and united States government approval is expensive and slow. Not something that works well with a degenerative disease.
I'm a bit insane right now admittedly, but given my background and wealth, I'm considering DIY. The technology is there, government is the hold up. Would you let your kid have seizures and cognitive problems because of red tape?
I think it's different