AAV gene therapy for GBA1-related diseases.
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| Title: | AAV gene therapy for GBA1-related diseases. |
|---|---|
| Authors: | Ayloo S; Genomic Medicine Unit, Sanofi, Waltham, MA 02451, USA., Ryu JC; Genomic Medicine Unit, Sanofi, Waltham, MA 02451, USA., Chou SC; Genomic Medicine Unit, Sanofi, Waltham, MA 02451, USA., Blatnik L; Genomic Medicine Unit, Sanofi, Waltham, MA 02451, USA., Wischhof E; Genomic Medicine Unit, Sanofi, Waltham, MA 02451, USA., Bu J; Genomic Medicine Unit, Sanofi, Waltham, MA 02451, USA., Guo L; Translational Medicine Unit, Sanofi, Cambridge, MA 02141, USA., Hossain M; Translational Medicine Unit, Sanofi, Cambridge, MA 02141, USA., Ghosh D; Translational Medicine Unit, Sanofi, Cambridge, MA 02141, USA., McCarty W; Quantitative Pharmacology, Sanofi, Cambridge, MA 02141, USA., Byrne A; Target Disease and Systems Biology, Cambridge, MA 02141, USA., Chai L; Target Disease and Systems Biology, Cambridge, MA 02141, USA., Clarke J; Rare Diseases TA, Sanofi, Cambridge, MA 02141, USA., Kayatekin C; Rare Diseases TA, Sanofi, Cambridge, MA 02141, USA., Zhang B; Translational Medicine Unit, Sanofi, Cambridge, MA 02141, USA., Sardi SP; Rare Diseases TA, Sanofi, Cambridge, MA 02141, USA., Goulet M; Genomic Medicine Unit, Sanofi, Waltham, MA 02451, USA., Elmer B; Genomic Medicine Unit, Sanofi, Waltham, MA 02451, USA., Mueller C; Genomic Medicine Unit, Sanofi, Waltham, MA 02451, USA. Electronic address: gmu@sanofi.com., Ramachandran S; Genomic Medicine Unit, Sanofi, Waltham, MA 02451, USA. |
| Source: | Molecular therapy : the journal of the American Society of Gene Therapy [Mol Ther] 2026 Mar 04; Vol. 34 (3), pp. 1707-1728. Date of Electronic Publication: 2025 Dec 09. |
| 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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