Preclinical evaluation of antisense oligonucleotide therapy in a mouse model of HNRNPH2-related neurodevelopmental disorder.
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| Title: | Preclinical evaluation of antisense oligonucleotide therapy in a mouse model of HNRNPH2-related neurodevelopmental disorder. |
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| Authors: | Korff A; Department of Cell and Molecular Biology, St. Jude Children's Research Hospital, Memphis, TN 38105, USA., Yang X; Department of Cell and Molecular Biology, St. Jude Children's Research Hospital, Memphis, TN 38105, USA., Ozdemir O; Department of Cell and Molecular Biology, St. Jude Children's Research Hospital, Memphis, TN 38105, USA., Samanta A; Department of Cell and Molecular Biology, St. Jude Children's Research Hospital, Memphis, TN 38105, USA., Wang YD; Department of Cell and Molecular Biology, St. Jude Children's Research Hospital, Memphis, TN 38105, USA., Patni T; Department of Biostatistics, St. Jude Children's Research Hospital, Memphis, TN 38105, USA., Lavado AJ; Center for Pediatric Neurological Disease Research, Pediatric Translational Neuroscience Initiative, St. Jude Children's Research Hospital, Memphis, TN 38105, USA., Kavirayani AM; Department of Pathology, St. Jude Children's Research Hospital, Memphis, TN 38105, USA., Ochaba J; Ionis Pharmaceuticals, Carlsbad, CA 92010, USA., Powers B; Ionis Pharmaceuticals, Carlsbad, CA 92010, USA., Bennett CF; Ionis Pharmaceuticals, Carlsbad, CA 92010, USA., Kim HJ; Department of Cell and Molecular Biology, St. Jude Children's Research Hospital, Memphis, TN 38105, USA., Taylor JP; Department of Cell and Molecular Biology, St. Jude Children's Research Hospital, Memphis, TN 38105, USA. |
| Source: | Science translational medicine [Sci Transl Med] 2026 Apr 22; Vol. 18 (846), pp. eadx3491. Date of Electronic Publication: 2026 Apr 22. |
| Publication Type: | Journal Article |
| Journal Info: | Publisher: American Association for the Advancement of Science Country of Publication: United States NLM ID: 101505086 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1946-6242 (Electronic) Linking ISSN: 19466234 NLM ISO Abbreviation: Sci Transl Med Subsets: MEDLINE |
| Database: | MEDLINE Ultimate |
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| ISSN: | 1946-6242 |
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| DOI: | 10.1126/scitranslmed.adx3491 |
