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. |
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| 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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