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AAV delivery of full-length SYNGAP1 rescues epileptic and behavioral phenotypes in a mouse model of SYNGAP1-related disorders.

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Title: AAV delivery of full-length SYNGAP1 rescues epileptic and behavioral phenotypes in a mouse model of SYNGAP1-related disorders.
Authors: Quinlan MA; Allen Institute for Brain Science, Seattle, WA 98109, USA., Guo R; Allen Institute for Brain Science, Seattle, WA 98109, USA., Clark AG; Allen Institute for Brain Science, Seattle, WA 98109, USA., Luber EM; Allen Institute for Brain Science, Seattle, WA 98109, USA., Christian RJ; Allen Institute for Brain Science, Seattle, WA 98109, USA., Martinez RA; Allen Institute for Brain Science, Seattle, WA 98109, USA., Groce EL; Allen Institute for Brain Science, Seattle, WA 98109, USA., Liu J; Allen Institute for Brain Science, Seattle, WA 98109, USA., Bishaw YM; Allen Institute for Brain Science, Seattle, WA 98109, USA., Bhowmik R; Allen Institute for Brain Science, Seattle, WA 98109, USA., Liang E; Allen Institute for Brain Science, Seattle, WA 98109, USA., Reding M; Allen Institute for Brain Science, Seattle, WA 98109, USA., Ronellenfitch K; Allen Institute for Brain Science, Seattle, WA 98109, USA., Wright V; Allen Institute for Brain Science, Seattle, WA 98109, USA., Gudsnuk KM; Allen Institute for Brain Science, Seattle, WA 98109, USA., Leedy JM; BioMarin Pharmaceutical, San Rafael, CA 94902, USA., Mich JK; Allen Institute for Brain Science, Seattle, WA 98109, USA., Gore BB; Allen Institute for Brain Science, Seattle, WA 98109, USA., Daigle TL; Allen Institute for Brain Science, Seattle, WA 98109, USA; Department of Neurobiology and Biophysics, University of Washington, Seattle, WA 98195, USA., Lopez ME; BioMarin Pharmaceutical, San Rafael, CA 94902, USA., Lein ES; Allen Institute for Brain Science, Seattle, WA 98109, USA; Department of Neurological Surgery, University of Washington, Seattle, WA 98195, USA., Ichida JK; BioMarin Pharmaceutical, San Rafael, CA 94902, USA., Levi BP; Allen Institute for Brain Science, Seattle, WA 98109, USA. Electronic address: boazl@alleninstitute.org.
Source: Molecular therapy : the journal of the American Society of Gene Therapy [Mol Ther] 2025 Dec 03; Vol. 33 (12), pp. 6398-6414. Date of Electronic Publication: 2025 Sep 23.
Publication Type: Journal Article
Journal Info: Publisher: Cell Press Country of Publication: United States NLM ID: 100890581 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1525-0024 (Electronic) Linking ISSN: 15250016 NLM ISO Abbreviation: Mol Ther Subsets: MEDLINE
Database: MEDLINE Ultimate
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PubType: Academic Journal
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  Data: AAV delivery of full-length SYNGAP1 rescues epileptic and behavioral phenotypes in a mouse model of SYNGAP1-related disorders.
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  Data: <searchLink fieldCode="AU" term="%22Quinlan+MA%22">Quinlan MA</searchLink>; Allen Institute for Brain Science, Seattle, WA 98109, USA.<br /><searchLink fieldCode="AU" term="%22Guo+R%22">Guo R</searchLink>; Allen Institute for Brain Science, Seattle, WA 98109, USA.<br /><searchLink fieldCode="AU" term="%22Clark+AG%22">Clark AG</searchLink>; Allen Institute for Brain Science, Seattle, WA 98109, USA.<br /><searchLink fieldCode="AU" term="%22Luber+EM%22">Luber EM</searchLink>; Allen Institute for Brain Science, Seattle, WA 98109, USA.<br /><searchLink fieldCode="AU" term="%22Christian+RJ%22">Christian RJ</searchLink>; Allen Institute for Brain Science, Seattle, WA 98109, USA.<br /><searchLink fieldCode="AU" term="%22Martinez+RA%22">Martinez RA</searchLink>; Allen Institute for Brain Science, Seattle, WA 98109, USA.<br /><searchLink fieldCode="AU" term="%22Groce+EL%22">Groce EL</searchLink>; Allen Institute for Brain Science, Seattle, WA 98109, USA.<br /><searchLink fieldCode="AU" term="%22Liu+J%22">Liu J</searchLink>; Allen Institute for Brain Science, Seattle, WA 98109, USA.<br /><searchLink fieldCode="AU" term="%22Bishaw+YM%22">Bishaw YM</searchLink>; Allen Institute for Brain Science, Seattle, WA 98109, USA.<br /><searchLink fieldCode="AU" term="%22Bhowmik+R%22">Bhowmik R</searchLink>; Allen Institute for Brain Science, Seattle, WA 98109, USA.<br /><searchLink fieldCode="AU" term="%22Liang+E%22">Liang E</searchLink>; Allen Institute for Brain Science, Seattle, WA 98109, USA.<br /><searchLink fieldCode="AU" term="%22Reding+M%22">Reding M</searchLink>; Allen Institute for Brain Science, Seattle, WA 98109, USA.<br /><searchLink fieldCode="AU" term="%22Ronellenfitch+K%22">Ronellenfitch K</searchLink>; Allen Institute for Brain Science, Seattle, WA 98109, USA.<br /><searchLink fieldCode="AU" term="%22Wright+V%22">Wright V</searchLink>; Allen Institute for Brain Science, Seattle, WA 98109, USA.<br /><searchLink fieldCode="AU" term="%22Gudsnuk+KM%22">Gudsnuk KM</searchLink>; Allen Institute for Brain Science, Seattle, WA 98109, USA.<br /><searchLink fieldCode="AU" term="%22Leedy+JM%22">Leedy JM</searchLink>; BioMarin Pharmaceutical, San Rafael, CA 94902, USA.<br /><searchLink fieldCode="AU" term="%22Mich+JK%22">Mich JK</searchLink>; Allen Institute for Brain Science, Seattle, WA 98109, USA.<br /><searchLink fieldCode="AU" term="%22Gore+BB%22">Gore BB</searchLink>; Allen Institute for Brain Science, Seattle, WA 98109, USA.<br /><searchLink fieldCode="AU" term="%22Daigle+TL%22">Daigle TL</searchLink>; Allen Institute for Brain Science, Seattle, WA 98109, USA; Department of Neurobiology and Biophysics, University of Washington, Seattle, WA 98195, USA.<br /><searchLink fieldCode="AU" term="%22Lopez+ME%22">Lopez ME</searchLink>; BioMarin Pharmaceutical, San Rafael, CA 94902, USA.<br /><searchLink fieldCode="AU" term="%22Lein+ES%22">Lein ES</searchLink>; Allen Institute for Brain Science, Seattle, WA 98109, USA; Department of Neurological Surgery, University of Washington, Seattle, WA 98195, USA.<br /><searchLink fieldCode="AU" term="%22Ichida+JK%22">Ichida JK</searchLink>; BioMarin Pharmaceutical, San Rafael, CA 94902, USA.<br /><searchLink fieldCode="AU" term="%22Levi+BP%22">Levi BP</searchLink>; Allen Institute for Brain Science, Seattle, WA 98109, USA. Electronic address: boazl@alleninstitute.org.
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  Data: <searchLink fieldCode="JN" term="%22100890581%22">Molecular therapy : the journal of the American Society of Gene Therapy</searchLink> [Mol Ther] 2025 Dec 03; Vol. 33 (12), pp. 6398-6414. <i>Date of Electronic Publication: </i>2025 Sep 23.
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  Data: <i>Publisher: </i><searchLink fieldCode="PB" term="%22Cell+Press%22">Cell Press </searchLink><i>Country of Publication: </i>United States <i>NLM ID: </i>100890581 <i>Publication Model: </i>Print-Electronic <i>Cited Medium: </i>Internet <i>ISSN: </i>1525-0024 (Electronic) <i>Linking ISSN: </i><searchLink fieldCode="IS" term="%2215250016%22">15250016 </searchLink><i>NLM ISO Abbreviation: </i>Mol Ther <i>Subsets: </i>MEDLINE
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        Value: 10.1016/j.ymthe.2025.09.040
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              Text: 2025 Dec 03
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