Single-dose administration of therapeutic divalent siRNA targeting MECP2 prevents lethality for one year in an MECP2 duplication mouse model
Vignesh Narayan Hariharan, Ashley Summers, Amy E. Clipperton‐Allen, Jillian Caiazzi, Samuel R. Hildebrand, Daniel O’Reilly, Qi Tang, Zachary Kennedy, Dimas Echeverria, Nicholas McHugh, David Cooper, Jacquelyn Sousa, Chantal Ferguson, Hassan H. Fakih, Laurent P. Bogdanik, , Anastasia Khvorova
Therapeutic siRNAs provide safe and durable modulation of MECP2 for the treatment of mild and severe MECP2 Duplication Syndrome.
Nature Communications · Jul 21, 2026Peer-reviewedNeuroscience