Review
Open access
Jul 2026
MeCP2 Dosage Control in Rett Syndrome: Non-Coding RNA-Based and Epigenetic Strategies for Safer Gene Therapy
Clinically applicable gene therapy for RTT will likely need to move beyond simple MECP2 replacement and instead rely on precise cell- and dose-dependent regulation of its expression, with an emphasis on non-coding RNA-based and epigenetic mechanisms.
I. Kabdesh, A. Rizvanov, Y. Mukhamedshina
· Non-Coding RNA · 0 citations