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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.
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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  Data: Preclinical evaluation of antisense oligonucleotide therapy in a mouse model of HNRNPH2-related neurodevelopmental disorder.
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  Data: <searchLink fieldCode="AU" term="%22Korff+A%22">Korff A</searchLink>; Department of Cell and Molecular Biology, St. Jude Children's Research Hospital, Memphis, TN 38105, USA.<br /><searchLink fieldCode="AU" term="%22Yang+X%22">Yang X</searchLink>; Department of Cell and Molecular Biology, St. Jude Children's Research Hospital, Memphis, TN 38105, USA.<br /><searchLink fieldCode="AU" term="%22Ozdemir+O%22">Ozdemir O</searchLink>; Department of Cell and Molecular Biology, St. Jude Children's Research Hospital, Memphis, TN 38105, USA.<br /><searchLink fieldCode="AU" term="%22Samanta+A%22">Samanta A</searchLink>; Department of Cell and Molecular Biology, St. Jude Children's Research Hospital, Memphis, TN 38105, USA.<br /><searchLink fieldCode="AU" term="%22Wang+YD%22">Wang YD</searchLink>; Department of Cell and Molecular Biology, St. Jude Children's Research Hospital, Memphis, TN 38105, USA.<br /><searchLink fieldCode="AU" term="%22Patni+T%22">Patni T</searchLink>; Department of Biostatistics, St. Jude Children's Research Hospital, Memphis, TN 38105, USA.<br /><searchLink fieldCode="AU" term="%22Lavado+AJ%22">Lavado AJ</searchLink>; Center for Pediatric Neurological Disease Research, Pediatric Translational Neuroscience Initiative, St. Jude Children's Research Hospital, Memphis, TN 38105, USA.<br /><searchLink fieldCode="AU" term="%22Kavirayani+AM%22">Kavirayani AM</searchLink>; Department of Pathology, St. Jude Children's Research Hospital, Memphis, TN 38105, USA.<br /><searchLink fieldCode="AU" term="%22Ochaba+J%22">Ochaba J</searchLink>; Ionis Pharmaceuticals, Carlsbad, CA 92010, USA.<br /><searchLink fieldCode="AU" term="%22Powers+B%22">Powers B</searchLink>; Ionis Pharmaceuticals, Carlsbad, CA 92010, USA.<br /><searchLink fieldCode="AU" term="%22Bennett+CF%22">Bennett CF</searchLink>; Ionis Pharmaceuticals, Carlsbad, CA 92010, USA.<br /><searchLink fieldCode="AU" term="%22Kim+HJ%22">Kim HJ</searchLink>; Department of Cell and Molecular Biology, St. Jude Children's Research Hospital, Memphis, TN 38105, USA.<br /><searchLink fieldCode="AU" term="%22Taylor+JP%22">Taylor JP</searchLink>; Department of Cell and Molecular Biology, St. Jude Children's Research Hospital, Memphis, TN 38105, USA.
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  Data: <searchLink fieldCode="JN" term="%22101505086%22">Science translational medicine</searchLink> [Sci Transl Med] 2026 Apr 22; Vol. 18 (846), pp. eadx3491. <i>Date of Electronic Publication: </i>2026 Apr 22.
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