Genetic Editing Milestone in Mouse Model of Rett Syndrome
Retrieved on:
Wednesday, July 15, 2020
The new research, published July 14 in the journal Cell Reports, shows that RNA editing may repair the underlying cause of Rett Syndrome in a mouse model.
Key Points:
- The new research, published July 14 in the journal Cell Reports, shows that RNA editing may repair the underlying cause of Rett Syndrome in a mouse model.
- The results represent a promising early step in using RNA editing to treat Rett Syndrome, a disorder that affects about 350,000 individuals worldwide.
- "The study is the first example of RNA editing in a mouse model of a neurological disease, and therefore a considerable step forward in the potential of RNA editing becoming a therapeutic for Rett Syndrome," says Monica Coenraads, executive director of Rett Syndrome Research Trust, which helped fund the study.
- The Rett Syndrome Research Trust (RSRT) is a non-profit organization with a highly personal and urgent mission: a cure for Rett Syndrome and related MECP2 disorders.