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CRISPR activation for SCN2A-related neurodevelopmental disorders.

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Title: CRISPR activation for SCN2A-related neurodevelopmental disorders.
Authors: Tamura S; Department of Bioengineering and Therapeutic Sciences, University of California San Francisco, San Francisco, CA, USA.; Institute for Human Genetics, University of California San Francisco, San Francisco, CA, USA., Nelson AD; Weill Institute for Neurosciences, University of California San Francisco, San Francisco, CA, USA.; Department of Neurology, University of California San Francisco, San Francisco, CA, USA., Spratt PWE; Weill Institute for Neurosciences, University of California San Francisco, San Francisco, CA, USA.; Department of Neurology, University of California San Francisco, San Francisco, CA, USA., Hamada EC; Weill Institute for Neurosciences, University of California San Francisco, San Francisco, CA, USA.; Department of Neurology, University of California San Francisco, San Francisco, CA, USA., Zhou X; Department of Bioengineering and Therapeutic Sciences, University of California San Francisco, San Francisco, CA, USA.; Institute for Human Genetics, University of California San Francisco, San Francisco, CA, USA., Kyoung H; Weill Institute for Neurosciences, University of California San Francisco, San Francisco, CA, USA.; Department of Neurology, University of California San Francisco, San Francisco, CA, USA., Li Z; Department of Bioengineering and Therapeutic Sciences, University of California San Francisco, San Francisco, CA, USA.; Institute for Human Genetics, University of California San Francisco, San Francisco, CA, USA., Arnould C; Department of Bioengineering and Therapeutic Sciences, University of California San Francisco, San Francisco, CA, USA.; Institute for Human Genetics, University of California San Francisco, San Francisco, CA, USA., Barskyi V; Department of Bioengineering and Therapeutic Sciences, University of California San Francisco, San Francisco, CA, USA.; Institute for Human Genetics, University of California San Francisco, San Francisco, CA, USA., Krupkin B; Department of Bioengineering and Therapeutic Sciences, University of California San Francisco, San Francisco, CA, USA.; Institute for Human Genetics, University of California San Francisco, San Francisco, CA, USA., Young K; Department of Bioengineering and Therapeutic Sciences, University of California San Francisco, San Francisco, CA, USA.; Institute for Human Genetics, University of California San Francisco, San Francisco, CA, USA., Zhao J; Department of Bioengineering and Therapeutic Sciences, University of California San Francisco, San Francisco, CA, USA.; Institute for Human Genetics, University of California San Francisco, San Francisco, CA, USA., Holden SS; Department of Neurology, University of California San Francisco, San Francisco, CA, USA.; Gladstone Institute of Neurological Disease, University of California San Francisco, San Francisco, CA, USA., Sahagun A; Weill Institute for Neurosciences, University of California San Francisco, San Francisco, CA, USA.; Department of Neurology, University of California San Francisco, San Francisco, CA, USA., Keeshen CM; Weill Institute for Neurosciences, University of California San Francisco, San Francisco, CA, USA.; Department of Neurology, University of California San Francisco, San Francisco, CA, USA., Lu C; Stanley Center for Psychiatric Research, Broad Institute, Cambridge, MA, USA., Ben-Shalom R; Weill Institute for Neurosciences, University of California San Francisco, San Francisco, CA, USA.; Department of Neurology, University of California San Francisco, San Francisco, CA, USA., Taloma SE; Weill Institute for Neurosciences, University of California San Francisco, San Francisco, CA, USA.; Department of Neurology, University of California San Francisco, San Francisco, CA, USA., Schamiloglu S; Weill Institute for Neurosciences, University of California San Francisco, San Francisco, CA, USA.; Department of Neurology, University of California San Francisco, San Francisco, CA, USA., Li YC; Weill Institute for Neurosciences, University of California San Francisco, San Francisco, CA, USA.; Department of Neurology, University of California San Francisco, San Francisco, CA, USA., Min L; Department of Pharmacology, University of Michigan Medical School, Ann Arbor, MI, USA., Jenkins PM; Department of Pharmacology, University of Michigan Medical School, Ann Arbor, MI, USA.; Department of Psychiatry, University of Michigan Medical School, Ann Arbor, MI, USA., Pan JQ; Stanley Center for Psychiatric Research, Broad Institute, Cambridge, MA, USA., Paz JT; Department of Neurology, University of California San Francisco, San Francisco, CA, USA.; Gladstone Institute of Neurological Disease, University of California San Francisco, San Francisco, CA, USA., Sanders SJ; Weill Institute for Neurosciences, University of California San Francisco, San Francisco, CA, USA.; Department of Psychiatry, University of California San Francisco, San Francisco, CA, USA.; Institute of Developmental and Regenerative Medicine, Department of Paediatrics, University of Oxford, Oxford, UK., Matharu N; Department of Bioengineering and Therapeutic Sciences, University of California San Francisco, San Francisco, CA, USA.; Institute for Human Genetics, University of California San Francisco, San Francisco, CA, USA., Ahituv N; Department of Bioengineering and Therapeutic Sciences, University of California San Francisco, San Francisco, CA, USA. nadav.ahituv@ucsf.edu.; Institute for Human Genetics, University of California San Francisco, San Francisco, CA, USA. nadav.ahituv@ucsf.edu., Bender KJ; Weill Institute for Neurosciences, University of California San Francisco, San Francisco, CA, USA. kevin.bender@ucsf.edu.; Department of Neurology, University of California San Francisco, San Francisco, CA, USA. kevin.bender@ucsf.edu.
Source: Nature [Nature] 2025 Oct; Vol. 646 (8086), pp. 983-991. Date of Electronic Publication: 2025 Sep 17.
Publication Type: Journal Article; Research Support, N.I.H., Extramural; Research Support, Non-U.S. Gov't
Journal Info: Publisher: Nature Publishing Group Country of Publication: England NLM ID: 0410462 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1476-4687 (Electronic) Linking ISSN: 00280836 NLM ISO Abbreviation: Nature Subsets: MEDLINE
Database: MEDLINE Ultimate
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  Data: CRISPR activation for SCN2A-related neurodevelopmental disorders.
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  Data: <searchLink fieldCode="AU" term="%22Tamura+S%22">Tamura S</searchLink>; Department of Bioengineering and Therapeutic Sciences, University of California San Francisco, San Francisco, CA, USA.; Institute for Human Genetics, University of California San Francisco, San Francisco, CA, USA.<br /><searchLink fieldCode="AU" term="%22Nelson+AD%22">Nelson AD</searchLink>; Weill Institute for Neurosciences, University of California San Francisco, San Francisco, CA, USA.; Department of Neurology, University of California San Francisco, San Francisco, CA, USA.<br /><searchLink fieldCode="AU" term="%22Spratt+PWE%22">Spratt PWE</searchLink>; Weill Institute for Neurosciences, University of California San Francisco, San Francisco, CA, USA.; Department of Neurology, University of California San Francisco, San Francisco, CA, USA.<br /><searchLink fieldCode="AU" term="%22Hamada+EC%22">Hamada EC</searchLink>; Weill Institute for Neurosciences, University of California San Francisco, San Francisco, CA, USA.; Department of Neurology, University of California San Francisco, San Francisco, CA, USA.<br /><searchLink fieldCode="AU" term="%22Zhou+X%22">Zhou X</searchLink>; Department of Bioengineering and Therapeutic Sciences, University of California San Francisco, San Francisco, CA, USA.; Institute for Human Genetics, University of California San Francisco, San Francisco, CA, USA.<br /><searchLink fieldCode="AU" term="%22Kyoung+H%22">Kyoung H</searchLink>; Weill Institute for Neurosciences, University of California San Francisco, San Francisco, CA, USA.; Department of Neurology, University of California San Francisco, San Francisco, CA, USA.<br /><searchLink fieldCode="AU" term="%22Li+Z%22">Li Z</searchLink>; Department of Bioengineering and Therapeutic Sciences, University of California San Francisco, San Francisco, CA, USA.; Institute for Human Genetics, University of California San Francisco, San Francisco, CA, USA.<br /><searchLink fieldCode="AU" term="%22Arnould+C%22">Arnould C</searchLink>; Department of Bioengineering and Therapeutic Sciences, University of California San Francisco, San Francisco, CA, USA.; Institute for Human Genetics, University of California San Francisco, San Francisco, CA, USA.<br /><searchLink fieldCode="AU" term="%22Barskyi+V%22">Barskyi V</searchLink>; Department of Bioengineering and Therapeutic Sciences, University of California San Francisco, San Francisco, CA, USA.; Institute for Human Genetics, University of California San Francisco, San Francisco, CA, USA.<br /><searchLink fieldCode="AU" term="%22Krupkin+B%22">Krupkin B</searchLink>; Department of Bioengineering and Therapeutic Sciences, University of California San Francisco, San Francisco, CA, USA.; Institute for Human Genetics, University of California San Francisco, San Francisco, CA, USA.<br /><searchLink fieldCode="AU" term="%22Young+K%22">Young K</searchLink>; Department of Bioengineering and Therapeutic Sciences, University of California San Francisco, San Francisco, CA, USA.; Institute for Human Genetics, University of California San Francisco, San Francisco, CA, USA.<br /><searchLink fieldCode="AU" term="%22Zhao+J%22">Zhao J</searchLink>; Department of Bioengineering and Therapeutic Sciences, University of California San Francisco, San Francisco, CA, USA.; Institute for Human Genetics, University of California San Francisco, San Francisco, CA, USA.<br /><searchLink fieldCode="AU" term="%22Holden+SS%22">Holden SS</searchLink>; Department of Neurology, University of California San Francisco, San Francisco, CA, USA.; Gladstone Institute of Neurological Disease, University of California San Francisco, San Francisco, CA, USA.<br /><searchLink fieldCode="AU" term="%22Sahagun+A%22">Sahagun A</searchLink>; Weill Institute for Neurosciences, University of California San Francisco, San Francisco, CA, USA.; Department of Neurology, University of California San Francisco, San Francisco, CA, USA.<br /><searchLink fieldCode="AU" term="%22Keeshen+CM%22">Keeshen CM</searchLink>; Weill Institute for Neurosciences, University of California San Francisco, San Francisco, CA, USA.; Department of Neurology, University of California San Francisco, San Francisco, CA, USA.<br /><searchLink fieldCode="AU" term="%22Lu+C%22">Lu C</searchLink>; Stanley Center for Psychiatric Research, Broad Institute, Cambridge, MA, USA.<br /><searchLink fieldCode="AU" term="%22Ben-Shalom+R%22">Ben-Shalom R</searchLink>; Weill Institute for Neurosciences, University of California San Francisco, San Francisco, CA, USA.; Department of Neurology, University of California San Francisco, San Francisco, CA, USA.<br /><searchLink fieldCode="AU" term="%22Taloma+SE%22">Taloma SE</searchLink>; Weill Institute for Neurosciences, University of California San Francisco, San Francisco, CA, USA.; Department of Neurology, University of California San Francisco, San Francisco, CA, USA.<br /><searchLink fieldCode="AU" term="%22Schamiloglu+S%22">Schamiloglu S</searchLink>; Weill Institute for Neurosciences, University of California San Francisco, San Francisco, CA, USA.; Department of Neurology, University of California San Francisco, San Francisco, CA, USA.<br /><searchLink fieldCode="AU" term="%22Li+YC%22">Li YC</searchLink>; Weill Institute for Neurosciences, University of California San Francisco, San Francisco, CA, USA.; Department of Neurology, University of California San Francisco, San Francisco, CA, USA.<br /><searchLink fieldCode="AU" term="%22Min+L%22">Min L</searchLink>; Department of Pharmacology, University of Michigan Medical School, Ann Arbor, MI, USA.<br /><searchLink fieldCode="AU" term="%22Jenkins+PM%22">Jenkins PM</searchLink>; Department of Pharmacology, University of Michigan Medical School, Ann Arbor, MI, USA.; Department of Psychiatry, University of Michigan Medical School, Ann Arbor, MI, USA.<br /><searchLink fieldCode="AU" term="%22Pan+JQ%22">Pan JQ</searchLink>; Stanley Center for Psychiatric Research, Broad Institute, Cambridge, MA, USA.<br /><searchLink fieldCode="AU" term="%22Paz+JT%22">Paz JT</searchLink>; Department of Neurology, University of California San Francisco, San Francisco, CA, USA.; Gladstone Institute of Neurological Disease, University of California San Francisco, San Francisco, CA, USA.<br /><searchLink fieldCode="AU" term="%22Sanders+SJ%22">Sanders SJ</searchLink>; Weill Institute for Neurosciences, University of California San Francisco, San Francisco, CA, USA.; Department of Psychiatry, University of California San Francisco, San Francisco, CA, USA.; Institute of Developmental and Regenerative Medicine, Department of Paediatrics, University of Oxford, Oxford, UK.<br /><searchLink fieldCode="AU" term="%22Matharu+N%22">Matharu N</searchLink>; Department of Bioengineering and Therapeutic Sciences, University of California San Francisco, San Francisco, CA, USA.; Institute for Human Genetics, University of California San Francisco, San Francisco, CA, USA.<br /><searchLink fieldCode="AU" term="%22Ahituv+N%22">Ahituv N</searchLink>; Department of Bioengineering and Therapeutic Sciences, University of California San Francisco, San Francisco, CA, USA. nadav.ahituv@ucsf.edu.; Institute for Human Genetics, University of California San Francisco, San Francisco, CA, USA. nadav.ahituv@ucsf.edu.<br /><searchLink fieldCode="AU" term="%22Bender+KJ%22">Bender KJ</searchLink>; Weill Institute for Neurosciences, University of California San Francisco, San Francisco, CA, USA. kevin.bender@ucsf.edu.; Department of Neurology, University of California San Francisco, San Francisco, CA, USA. kevin.bender@ucsf.edu.
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  Data: <searchLink fieldCode="JN" term="%220410462%22">Nature</searchLink> [Nature] 2025 Oct; Vol. 646 (8086), pp. 983-991. <i>Date of Electronic Publication: </i>2025 Sep 17.
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