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Identification of a therapeutic threshold for AAV-STXBP1 gene therapy in a rodent model of STXBP1 developmental and epileptic encephalopathy.

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Title: Identification of a therapeutic threshold for AAV-STXBP1 gene therapy in a rodent model of STXBP1 developmental and epileptic encephalopathy.
Authors: Gallo NB; Biogen, Cambridge, MA 02142, USA., Annand JW; Biogen, Cambridge, MA 02142, USA., Clarner P; Biogen, Cambridge, MA 02142, USA., Harvey B; Biogen, Cambridge, MA 02142, USA., Morozova E; Biogen, Cambridge, MA 02142, USA., Ehrenfels C; Biogen, Cambridge, MA 02142, USA., Jiang T; Biogen, Cambridge, MA 02142, USA., Yuan L; Biogen, Cambridge, MA 02142, USA., Kashyap S; Biogen, Cambridge, MA 02142, USA., Lu X; Biogen, Cambridge, MA 02142, USA., Choi J; Biogen, Cambridge, MA 02142, USA., Zoll J; Biogen, Cambridge, MA 02142, USA., Nhu A; Biogen, Cambridge, MA 02142, USA., Brennecke A; Biogen, Cambridge, MA 02142, USA., Patel P; Biogen, Cambridge, MA 02142, USA., Gianni D; Biogen, Cambridge, MA 02142, USA., Guilmette E; Biogen, Cambridge, MA 02142, USA., Geisler S; Biogen, Cambridge, MA 02142, USA., Sheehy A; Biogen, Cambridge, MA 02142, USA., Ghaemi K; Biogen, Cambridge, MA 02142, USA., Benezra S; Biogen, Cambridge, MA 02142, USA., Ghoshal A; Biogen, Cambridge, MA 02142, USA., Liu Y; Biogen, Cambridge, MA 02142, USA., Carlile T; Biogen, Cambridge, MA 02142, USA., Cao S; Biogen, Cambridge, MA 02142, USA., Casey F; Biogen, Cambridge, MA 02142, USA., Koirala S; Biogen, Cambridge, MA 02142, USA., Wu S; Biogen, Cambridge, MA 02142, USA. Electronic address: sijia.wu@biogen.com.
Source: Molecular therapy : the journal of the American Society of Gene Therapy [Mol Ther] 2026 Jul 01; Vol. 34 (7), pp. 3763-3782. Date of Electronic Publication: 2026 Mar 24.
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: Identification of a therapeutic threshold for AAV-STXBP1 gene therapy in a rodent model of STXBP1 developmental and epileptic encephalopathy.
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  Data: <searchLink fieldCode="AU" term="%22Gallo+NB%22">Gallo NB</searchLink>; Biogen, Cambridge, MA 02142, USA.<br /><searchLink fieldCode="AU" term="%22Annand+JW%22">Annand JW</searchLink>; Biogen, Cambridge, MA 02142, USA.<br /><searchLink fieldCode="AU" term="%22Clarner+P%22">Clarner P</searchLink>; Biogen, Cambridge, MA 02142, USA.<br /><searchLink fieldCode="AU" term="%22Harvey+B%22">Harvey B</searchLink>; Biogen, Cambridge, MA 02142, USA.<br /><searchLink fieldCode="AU" term="%22Morozova+E%22">Morozova E</searchLink>; Biogen, Cambridge, MA 02142, USA.<br /><searchLink fieldCode="AU" term="%22Ehrenfels+C%22">Ehrenfels C</searchLink>; Biogen, Cambridge, MA 02142, USA.<br /><searchLink fieldCode="AU" term="%22Jiang+T%22">Jiang T</searchLink>; Biogen, Cambridge, MA 02142, USA.<br /><searchLink fieldCode="AU" term="%22Yuan+L%22">Yuan L</searchLink>; Biogen, Cambridge, MA 02142, USA.<br /><searchLink fieldCode="AU" term="%22Kashyap+S%22">Kashyap S</searchLink>; Biogen, Cambridge, MA 02142, USA.<br /><searchLink fieldCode="AU" term="%22Lu+X%22">Lu X</searchLink>; Biogen, Cambridge, MA 02142, USA.<br /><searchLink fieldCode="AU" term="%22Choi+J%22">Choi J</searchLink>; Biogen, Cambridge, MA 02142, USA.<br /><searchLink fieldCode="AU" term="%22Zoll+J%22">Zoll J</searchLink>; Biogen, Cambridge, MA 02142, USA.<br /><searchLink fieldCode="AU" term="%22Nhu+A%22">Nhu A</searchLink>; Biogen, Cambridge, MA 02142, USA.<br /><searchLink fieldCode="AU" term="%22Brennecke+A%22">Brennecke A</searchLink>; Biogen, Cambridge, MA 02142, USA.<br /><searchLink fieldCode="AU" term="%22Patel+P%22">Patel P</searchLink>; Biogen, Cambridge, MA 02142, USA.<br /><searchLink fieldCode="AU" term="%22Gianni+D%22">Gianni D</searchLink>; Biogen, Cambridge, MA 02142, USA.<br /><searchLink fieldCode="AU" term="%22Guilmette+E%22">Guilmette E</searchLink>; Biogen, Cambridge, MA 02142, USA.<br /><searchLink fieldCode="AU" term="%22Geisler+S%22">Geisler S</searchLink>; Biogen, Cambridge, MA 02142, USA.<br /><searchLink fieldCode="AU" term="%22Sheehy+A%22">Sheehy A</searchLink>; Biogen, Cambridge, MA 02142, USA.<br /><searchLink fieldCode="AU" term="%22Ghaemi+K%22">Ghaemi K</searchLink>; Biogen, Cambridge, MA 02142, USA.<br /><searchLink fieldCode="AU" term="%22Benezra+S%22">Benezra S</searchLink>; Biogen, Cambridge, MA 02142, USA.<br /><searchLink fieldCode="AU" term="%22Ghoshal+A%22">Ghoshal A</searchLink>; Biogen, Cambridge, MA 02142, USA.<br /><searchLink fieldCode="AU" term="%22Liu+Y%22">Liu Y</searchLink>; Biogen, Cambridge, MA 02142, USA.<br /><searchLink fieldCode="AU" term="%22Carlile+T%22">Carlile T</searchLink>; Biogen, Cambridge, MA 02142, USA.<br /><searchLink fieldCode="AU" term="%22Cao+S%22">Cao S</searchLink>; Biogen, Cambridge, MA 02142, USA.<br /><searchLink fieldCode="AU" term="%22Casey+F%22">Casey F</searchLink>; Biogen, Cambridge, MA 02142, USA.<br /><searchLink fieldCode="AU" term="%22Koirala+S%22">Koirala S</searchLink>; Biogen, Cambridge, MA 02142, USA.<br /><searchLink fieldCode="AU" term="%22Wu+S%22">Wu S</searchLink>; Biogen, Cambridge, MA 02142, USA. Electronic address: sijia.wu@biogen.com.
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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] 2026 Jul 01; Vol. 34 (7), pp. 3763-3782. <i>Date of Electronic Publication: </i>2026 Mar 24.
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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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