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In vivo genome editing using novel AAV-PHP variants rescues motor function deficits and extends survival in a SOD1-ALS mouse model.

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Title: In vivo genome editing using novel AAV-PHP variants rescues motor function deficits and extends survival in a SOD1-ALS mouse model.
Authors: Chen YA; Biogen Inc., Cambridge, MA, USA., Kankel MW; Apple Tree Partners (ATP) Research & Development, Branford, CT, USA., Hana S; Biogen Inc., Cambridge, MA, USA., Lau SK; Biogen Inc., Cambridge, MA, USA., Zavodszky MI; Biogen Inc., Cambridge, MA, USA., McKissick O; Biogen Inc., Cambridge, MA, USA., Mastrangelo N; Biogen Inc., Cambridge, MA, USA., Dion J; Biogen Inc., Cambridge, MA, USA., Wang B; Biogen Inc., Cambridge, MA, USA., Ferretti D; Biogen Inc., Cambridge, MA, USA., Koske D; Biogen Inc., Cambridge, MA, USA., Lehman S; Biogen Inc., Cambridge, MA, USA., Koszka K; Biogen Inc., Cambridge, MA, USA., McLaughlin H; Biogen Inc., Cambridge, MA, USA., Liu M; Biogen Inc., Cambridge, MA, USA., Marshall E; Biogen Inc., Cambridge, MA, USA., Fabian AJ; Biogen Inc., Cambridge, MA, USA., Cullen P; Biogen Inc., Cambridge, MA, USA., Marsh G; Biogen Inc., Cambridge, MA, USA., Hamann S; Biogen Inc., Cambridge, MA, USA., Craft M; Biogen Inc., Cambridge, MA, USA., Sebalusky J; Biogen Inc., Cambridge, MA, USA., Arnold HM; Biogen Inc., Cambridge, MA, USA., Driscoll R; Biogen Inc., Cambridge, MA, USA., Sheehy A; Biogen Inc., Cambridge, MA, USA., Luo Y; Biogen Inc., Cambridge, MA, USA., Manca S; Biogen Inc., Cambridge, MA, USA., Carlile T; Biogen Inc., Cambridge, MA, USA., Sun C; Biogen Inc., Cambridge, MA, USA., Sigrist K; Biogen Inc., Cambridge, MA, USA., McCampbell A; Biogen Inc., Cambridge, MA, USA., Henderson CE; Biogen Inc., Cambridge, MA, USA., Lo SC; Biogen Inc., Cambridge, MA, USA. joyce.lo@biogen.com.
Source: Gene therapy [Gene Ther] 2023 May; Vol. 30 (5), pp. 443-454. Date of Electronic Publication: 2022 Dec 01.
Publication Type: Journal Article; Research Support, Non-U.S. Gov't
Journal Info: Publisher: Nature Publishing Group Country of Publication: England NLM ID: 9421525 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1476-5462 (Electronic) Linking ISSN: 09697128 NLM ISO Abbreviation: Gene Ther Subsets: MEDLINE
Database: MEDLINE Ultimate
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  Data: In vivo genome editing using novel AAV-PHP variants rescues motor function deficits and extends survival in a SOD1-ALS mouse model.
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  Data: <searchLink fieldCode="AU" term="%22Chen+YA%22">Chen YA</searchLink>; Biogen Inc., Cambridge, MA, USA.<br /><searchLink fieldCode="AU" term="%22Kankel+MW%22">Kankel MW</searchLink>; Apple Tree Partners (ATP) Research & Development, Branford, CT, USA.<br /><searchLink fieldCode="AU" term="%22Hana+S%22">Hana S</searchLink>; Biogen Inc., Cambridge, MA, USA.<br /><searchLink fieldCode="AU" term="%22Lau+SK%22">Lau SK</searchLink>; Biogen Inc., Cambridge, MA, USA.<br /><searchLink fieldCode="AU" term="%22Zavodszky+MI%22">Zavodszky MI</searchLink>; Biogen Inc., Cambridge, MA, USA.<br /><searchLink fieldCode="AU" term="%22McKissick+O%22">McKissick O</searchLink>; Biogen Inc., Cambridge, MA, USA.<br /><searchLink fieldCode="AU" term="%22Mastrangelo+N%22">Mastrangelo N</searchLink>; Biogen Inc., Cambridge, MA, USA.<br /><searchLink fieldCode="AU" term="%22Dion+J%22">Dion J</searchLink>; Biogen Inc., Cambridge, MA, USA.<br /><searchLink fieldCode="AU" term="%22Wang+B%22">Wang B</searchLink>; Biogen Inc., Cambridge, MA, USA.<br /><searchLink fieldCode="AU" term="%22Ferretti+D%22">Ferretti D</searchLink>; Biogen Inc., Cambridge, MA, USA.<br /><searchLink fieldCode="AU" term="%22Koske+D%22">Koske D</searchLink>; Biogen Inc., Cambridge, MA, USA.<br /><searchLink fieldCode="AU" term="%22Lehman+S%22">Lehman S</searchLink>; Biogen Inc., Cambridge, MA, USA.<br /><searchLink fieldCode="AU" term="%22Koszka+K%22">Koszka K</searchLink>; Biogen Inc., Cambridge, MA, USA.<br /><searchLink fieldCode="AU" term="%22McLaughlin+H%22">McLaughlin H</searchLink>; Biogen Inc., Cambridge, MA, USA.<br /><searchLink fieldCode="AU" term="%22Liu+M%22">Liu M</searchLink>; Biogen Inc., Cambridge, MA, USA.<br /><searchLink fieldCode="AU" term="%22Marshall+E%22">Marshall E</searchLink>; Biogen Inc., Cambridge, MA, USA.<br /><searchLink fieldCode="AU" term="%22Fabian+AJ%22">Fabian AJ</searchLink>; Biogen Inc., Cambridge, MA, USA.<br /><searchLink fieldCode="AU" term="%22Cullen+P%22">Cullen P</searchLink>; Biogen Inc., Cambridge, MA, USA.<br /><searchLink fieldCode="AU" term="%22Marsh+G%22">Marsh G</searchLink>; Biogen Inc., Cambridge, MA, USA.<br /><searchLink fieldCode="AU" term="%22Hamann+S%22">Hamann S</searchLink>; Biogen Inc., Cambridge, MA, USA.<br /><searchLink fieldCode="AU" term="%22Craft+M%22">Craft M</searchLink>; Biogen Inc., Cambridge, MA, USA.<br /><searchLink fieldCode="AU" term="%22Sebalusky+J%22">Sebalusky J</searchLink>; Biogen Inc., Cambridge, MA, USA.<br /><searchLink fieldCode="AU" term="%22Arnold+HM%22">Arnold HM</searchLink>; Biogen Inc., Cambridge, MA, USA.<br /><searchLink fieldCode="AU" term="%22Driscoll+R%22">Driscoll R</searchLink>; Biogen Inc., Cambridge, MA, USA.<br /><searchLink fieldCode="AU" term="%22Sheehy+A%22">Sheehy A</searchLink>; Biogen Inc., Cambridge, MA, USA.<br /><searchLink fieldCode="AU" term="%22Luo+Y%22">Luo Y</searchLink>; Biogen Inc., Cambridge, MA, USA.<br /><searchLink fieldCode="AU" term="%22Manca+S%22">Manca S</searchLink>; Biogen Inc., Cambridge, MA, USA.<br /><searchLink fieldCode="AU" term="%22Carlile+T%22">Carlile T</searchLink>; Biogen Inc., Cambridge, MA, USA.<br /><searchLink fieldCode="AU" term="%22Sun+C%22">Sun C</searchLink>; Biogen Inc., Cambridge, MA, USA.<br /><searchLink fieldCode="AU" term="%22Sigrist+K%22">Sigrist K</searchLink>; Biogen Inc., Cambridge, MA, USA.<br /><searchLink fieldCode="AU" term="%22McCampbell+A%22">McCampbell A</searchLink>; Biogen Inc., Cambridge, MA, USA.<br /><searchLink fieldCode="AU" term="%22Henderson+CE%22">Henderson CE</searchLink>; Biogen Inc., Cambridge, MA, USA.<br /><searchLink fieldCode="AU" term="%22Lo+SC%22">Lo SC</searchLink>; Biogen Inc., Cambridge, MA, USA. joyce.lo@biogen.com.
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  Data: <searchLink fieldCode="JN" term="%229421525%22">Gene therapy</searchLink> [Gene Ther] 2023 May; Vol. 30 (5), pp. 443-454. <i>Date of Electronic Publication: </i>2022 Dec 01.
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  Data: <i>Publisher: </i><searchLink fieldCode="PB" term="%22Nature+Publishing+Group%22">Nature Publishing Group </searchLink><i>Country of Publication: </i>England <i>NLM ID: </i>9421525 <i>Publication Model: </i>Print-Electronic <i>Cited Medium: </i>Internet <i>ISSN: </i>1476-5462 (Electronic) <i>Linking ISSN: </i><searchLink fieldCode="IS" term="%2209697128%22">09697128 </searchLink><i>NLM ISO Abbreviation: </i>Gene Ther <i>Subsets: </i>MEDLINE
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