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. |
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| 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: | Research square [Res Sq] 2025 Apr 25. Date of Electronic Publication: 2025 Apr 25. |
| Publication Type: | Journal Article; Preprint |
| Journal Info: | Country of Publication: United States NLM ID: 101768035 Publication Model: Electronic Cited Medium: Internet ISSN: 2693-5015 (Electronic) Linking ISSN: 26935015 NLM ISO Abbreviation: Res Sq Subsets: PubMed not MEDLINE |
| Database: | MEDLINE Ultimate |
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