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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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Bibliographic Details
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
Description
ISSN:1525-0024
DOI:10.1016/j.ymthe.2025.09.040