Targeting neuronal lysosomal dysfunction caused by β-glucocerebrosidase deficiency with an enzyme-based brain shuttle construct.
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| Title: | Targeting neuronal lysosomal dysfunction caused by β-glucocerebrosidase deficiency with an enzyme-based brain shuttle construct. |
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| Authors: | Gehrlein A; Roche Pharma Research and Early Development, Neuroscience and Rare Diseases Discovery and Translational Area, Roche Innovation Center Basel, F. Hoffmann-La Roche Ltd, Basel, Switzerland. alexandra.gehrlein@roche.com., Udayar V; Roche Pharma Research and Early Development, Neuroscience and Rare Diseases Discovery and Translational Area, Roche Innovation Center Basel, F. Hoffmann-La Roche Ltd, Basel, Switzerland., Anastasi N; Roche Pharma Research and Early Development, Neuroscience and Rare Diseases Discovery and Translational Area, Roche Innovation Center Basel, F. Hoffmann-La Roche Ltd, Basel, Switzerland., Morella ML; Roche Pharma Research and Early Development, Neuroscience and Rare Diseases Discovery and Translational Area, Roche Innovation Center Basel, F. Hoffmann-La Roche Ltd, Basel, Switzerland.; Department of Anatomy and Neurosciences, Amsterdam University Medical Center | VUmc, Amsterdam, Netherlands., Ruf I; Roche Pharma Research and Early Development, Therapeutic Modalities, Lead Discovery, Roche Innovation Center Basel, F. Hoffmann-La Roche Ltd, Basel, Switzerland., Brugger D; Roche Pharma Research and Early Development, Therapeutic Modalities, Lead Discovery, Roche Innovation Center Basel, F. Hoffmann-La Roche Ltd, Basel, Switzerland., von der Mark S; Roche Pharma Research and Early Development, Therapeutic Modalities, Lead Discovery, Roche Innovation Center Basel, F. Hoffmann-La Roche Ltd, Basel, Switzerland., Thoma R; Roche Pharma Research and Early Development, Therapeutic Modalities, Lead Discovery, Roche Innovation Center Basel, F. Hoffmann-La Roche Ltd, Basel, Switzerland., Rufer A; Roche Pharma Research and Early Development, Therapeutic Modalities, Lead Discovery, Roche Innovation Center Basel, F. Hoffmann-La Roche Ltd, Basel, Switzerland., Heer D; Roche Pharma Research and Early Development, Therapeutic Modalities, Lead Discovery, Roche Innovation Center Basel, F. Hoffmann-La Roche Ltd, Basel, Switzerland., Pfahler N; Roche Pharma Research and Early Development, Therapeutic Modalities, Lead Discovery, Roche Innovation Center Basel, F. Hoffmann-La Roche Ltd, Basel, Switzerland.; Interfaculty Institute of Biochemistry & Structural Biology Biochemistry (IFIB), Eberhard Karls University of Tübingen, Tübingen, Germany., Jochner A; Roche Pharma Research and Early Development, Therapeutic Modalities Large Molecule Research, Roche Innovation Center Munich, Roche Diagnostics GmbH, Penzberg, Germany., Niewoehner J; Roche Pharma Research and Early Development, Therapeutic Modalities Large Molecule Research, Roche Innovation Center Munich, Roche Diagnostics GmbH, Penzberg, Germany., Wolf L; Roche Pharma Research and Early Development, Data & Analytics, Roche Innovation Center Basel, F. Hoffmann-La Roche Ltd, Basel, Switzerland., Fueth M; Roche Pharma Research and Early Development, Pharmaceutical Science, Roche Innovation Center Basel, F. Hoffmann-La Roche Ltd, Basel, Switzerland., Ebeling M; Roche Pharma Research and Early Development, Pharmaceutical Science, Roche Innovation Center Basel, F. Hoffmann-La Roche Ltd, Basel, Switzerland., Villaseñor R; Roche Pharma Research and Early Development, Neuroscience and Rare Diseases Discovery and Translational Area, Roche Innovation Center Basel, F. Hoffmann-La Roche Ltd, Basel, Switzerland., Zhu Y; Department of Chemistry, Simon Fraser University, Burnaby, BC, V5A 1S6, Canada., Deen MC; Department of Chemistry, Simon Fraser University, Burnaby, BC, V5A 1S6, Canada., Shan X; Department of Chemistry, Simon Fraser University, Burnaby, BC, V5A 1S6, Canada., Ehsaei Z; Department of Biomedicine, University of Basel, Basel, Switzerland., Taylor V; Department of Biomedicine, University of Basel, Basel, Switzerland., Sidransky E; Molecular Neurogenetics Section, National Human Genome Research Institute, Bethesda, MD, USA., Vocadlo DJ; Department of Chemistry, Simon Fraser University, Burnaby, BC, V5A 1S6, Canada.; Department of Molecular Biology and Biochemistry, Simon Fraser University, Burnaby, BC, V5A 1S6, Canada., Freskgård PO; Roche Pharma Research and Early Development, Neuroscience and Rare Diseases Discovery and Translational Area, Roche Innovation Center Basel, F. Hoffmann-La Roche Ltd, Basel, Switzerland.; BioArctic AB, Stockholm, Sweden., Jagasia R; Roche Pharma Research and Early Development, Neuroscience and Rare Diseases Discovery and Translational Area, Roche Innovation Center Basel, F. Hoffmann-La Roche Ltd, Basel, Switzerland. ravi.jagasia@roche.com. |
| Source: | Nature communications [Nat Commun] 2023 Apr 12; Vol. 14 (1), pp. 2057. Date of Electronic Publication: 2023 Apr 12. |
| Publication Type: | Journal Article |
| Journal Info: | Publisher: Nature Pub. Group Country of Publication: England NLM ID: 101528555 Publication Model: Electronic Cited Medium: Internet ISSN: 2041-1723 (Electronic) Linking ISSN: 20411723 NLM ISO Abbreviation: Nat Commun Subsets: MEDLINE |
| Database: | MEDLINE Ultimate |
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