University of Edinburgh
Gene Therapy Consortium 2.0
The Rett Syndrome Research Trust (RSRT) is a nonprofit organization with a highly personal and urgent mission: a
cure for Rett Syndrome and related MECP2 disorders. RSRT operates at the nexus of global scientific activity enabling advances in knowledge and driving innovative research. In March of 2017 RSRT announced Roadmap to a Cure, a three- year, $33 million strategic research plan. The plan, for which $24 million has been pledged or contributed by generous donors, prioritizes four curative approaches with gene therapy as our lead program. In June of 2017 the biotechnology company, AveXis, announced its intent to advance RSRT’s gene therapy program to clinical trials. Since 2008, RSRT has awarded $57 million to research. To learn more, please visit www.reverserett.org
Rett Syndrome is a genetic childhood neurological disorder caused by random mutations of the MECP2 gene on the X chromosome that affects predominately girls but can rarely also affect boys. Its symptoms typically become apparent between the ages of 12 to 18 months. Rett Syndrome is devastating as it deprives young children of speech, hand use, and normal movement often including the ability to walk. As the children enter childhood the disorder brings anxiety, seizures, tremors, breathing difficulties, and severe gastrointestinal issues. While their bodies suffer, it is believed that their cognitive abilities remain largely intact. Although most children survive to adulthood, they require total round- the-clock care.