Single-dose administration of therapeutic divalent siRNA targeting MECP2 prevents lethality in an MECP2 duplication mouse model.

Authors: Hariharan VN, Summers A, Clipperton-Allen AE, Caiazzi J, Hildebrand SR, O' Reilly D, Tang Q, Kennedy Z, Echeverria D, McHugh N, Cooper D, Sousa J, Ferguson C, Fakih HH, Bogdanik L, Coenraads M, Khvorova A

Journal: Nature communications