Girls with Rett Syndrome have a healthy copy of MECP2 on one X chromosome and a Rett-causing copy on the other X. Since each cell uses only one X, only one MECP2 gene is used to make protein – sometimes it’s the healthy copy and sometimes it’s the Rett-causing copy. If we could turn on the MECP2 gene on the inactive X, we would add in the healthy copy of MECP2 and rescue cells that are using the Rett-causing MECP2 gene, and theoretically, resolve the root cause of the disorder.
Register to learn about the progress being made by Drs. Kyle Fink and Antonio Bedalov to reactivate the back-up MECP2. Their labs recently received a $1.1 million award from RSRT.