At the core of the Rett Syndrome Research Trust (RSRT) Roadmap for a Cure, are four cutting-edge priority approaches that are designed to cure Rett Syndrome by attacking the root cause of the disorder: MECP2. These approaches, pursued in parallel, are applicable to all MECP2 mutations and deletions.

Gene Therapy

A single gene therapy treatment
could be a one-time fix

MECP2 Reactivation

Wake up healthy copies of
the MECP2 gene

RNA Modifications

Potential modification of the
gene at the RNA level

Protein Replacement

Bypass the gene to provide the
needed proteins to cells

*Used with the permission of RSRT


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