In March of this year we announced our strategic research plan, Roadmap to a Cure, the cornerstone of which is four therapeutic approaches that we are undertaking in pursuit of a cure for Rett: gene therapy, MECP2 Reactivation, RNA editing/splicing, and protein replacement.
Today’s announcement focuses on MECP2 Reactivation. A number of years ago we launched our MECP2 Reactivation Consortium which has primarily focused on screening for drugs that can reactivate the silenced MECP2. While this effort continues we are always on the lookout to leverage new technologies and resources.
We are pleased to welcome Rudolf Jaenisch and his lab colleagues to our MECP2 Reactivation effort. Watch the video below to see how Dr. Jaenisch will be using CRISPR to awaken MECP2.