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

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Title: Single-dose administration of therapeutic divalent siRNA targeting MECP2 prevents lethality for one year in an MECP2 duplication mouse model.
Authors: Hariharan VN; RNA Therapeutic Institute, University of Massachusetts Chan Medical School; Worcester, United States of America., Summers A; RNA Therapeutic Institute, University of Massachusetts Chan Medical School; Worcester, United States of America., Clipperton-Allen AE; The Jackson Laboratory; Bar Harbor, United States of America., Caiazzi J; RNA Therapeutic Institute, University of Massachusetts Chan Medical School; Worcester, United States of America., Hildebrand SR; RNA Therapeutic Institute, University of Massachusetts Chan Medical School; Worcester, United States of America., O'Reilly D; RNA Therapeutic Institute, University of Massachusetts Chan Medical School; Worcester, United States of America., Tang Q; RNA Therapeutic Institute, University of Massachusetts Chan Medical School; Worcester, United States of America., Kennedy Z; RNA Therapeutic Institute, University of Massachusetts Chan Medical School; Worcester, United States of America., Echeverria D; RNA Therapeutic Institute, University of Massachusetts Chan Medical School; Worcester, United States of America., McHugh N; RNA Therapeutic Institute, University of Massachusetts Chan Medical School; Worcester, United States of America., Cooper D; RNA Therapeutic Institute, University of Massachusetts Chan Medical School; Worcester, United States of America., Souza J; RNA Therapeutic Institute, University of Massachusetts Chan Medical School; Worcester, United States of America., Ferguson C; RNA Therapeutic Institute, University of Massachusetts Chan Medical School; Worcester, United States of America., Bogdanik L; The Jackson Laboratory; Bar Harbor, United States of America., Coenraads M; The Rett Syndrome Research Trust; Trumbull, United States of America., Khvorova A; RNA Therapeutic Institute, University of Massachusetts Chan Medical School; Worcester, United States of America.
Source: BioRxiv : the preprint server for biology [bioRxiv] 2025 Mar 29. Date of Electronic Publication: 2025 Mar 29.
Publication Type: Journal Article; Preprint
Journal Info: Country of Publication: United States NLM ID: 101680187 Publication Model: Electronic Cited Medium: Internet ISSN: 2692-8205 (Electronic) Linking ISSN: 26928205 NLM ISO Abbreviation: bioRxiv Subsets: PubMed not MEDLINE
Database: MEDLINE Ultimate
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  Data: Single-dose administration of therapeutic divalent siRNA targeting MECP2 prevents lethality for one year in an MECP2 duplication mouse model.
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  Data: <searchLink fieldCode="JN" term="%22101680187%22">BioRxiv : the preprint server for biology</searchLink> [bioRxiv] 2025 Mar 29. <i>Date of Electronic Publication: </i>2025 Mar 29.
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              Text: 2025 Mar 29
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