All of our Cure 360 strategies will require the delivery of therapeutics to the brain and, ideally, the ability to regulate how much of the MECP2 protein is made. Understanding how MECP2 functions informs the ability for regulation. Our scientific resources increase our foundational knowledge base, exponentially increasing our chances of success.
Enhancing delivery to the brain of Cure 360 therapeutics is one of our highest priorities.
If the need for tight protein regulation arises we’ll be ready with programs under development in the labs of Stuart Cobb and Michael Elowitz.
The Mandel and Bird labs showed, for the first time, a dramatic reversal of symptoms in fully symptomatic Rett mice using gene therapy techniques that could be used in people.
Attending the RSRT Consortium meetings is a wonderful experience. There is a collaborative atmosphere you don‘t see at large scientific meetings and everyone is focused on understanding the biology of MECP2 so that we can understand Rett Syndrome.
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.