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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.
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
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  Data: Targeting neuronal lysosomal dysfunction caused by β-glucocerebrosidase deficiency with an enzyme-based brain shuttle construct.
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  Data: <searchLink fieldCode="AU" term="%22Gehrlein+A%22">Gehrlein A</searchLink>; 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.<br /><searchLink fieldCode="AU" term="%22Udayar+V%22">Udayar V</searchLink>; 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.<br /><searchLink fieldCode="AU" term="%22Anastasi+N%22">Anastasi N</searchLink>; 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.<br /><searchLink fieldCode="AU" term="%22Morella+ML%22">Morella ML</searchLink>; 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.<br /><searchLink fieldCode="AU" term="%22Ruf+I%22">Ruf I</searchLink>; Roche Pharma Research and Early Development, Therapeutic Modalities, Lead Discovery, Roche Innovation Center Basel, F. Hoffmann-La Roche Ltd, Basel, Switzerland.<br /><searchLink fieldCode="AU" term="%22Brugger+D%22">Brugger D</searchLink>; Roche Pharma Research and Early Development, Therapeutic Modalities, Lead Discovery, Roche Innovation Center Basel, F. Hoffmann-La Roche Ltd, Basel, Switzerland.<br /><searchLink fieldCode="AU" term="%22von+der+Mark+S%22">von der Mark S</searchLink>; Roche Pharma Research and Early Development, Therapeutic Modalities, Lead Discovery, Roche Innovation Center Basel, F. Hoffmann-La Roche Ltd, Basel, Switzerland.<br /><searchLink fieldCode="AU" term="%22Thoma+R%22">Thoma R</searchLink>; Roche Pharma Research and Early Development, Therapeutic Modalities, Lead Discovery, Roche Innovation Center Basel, F. Hoffmann-La Roche Ltd, Basel, Switzerland.<br /><searchLink fieldCode="AU" term="%22Rufer+A%22">Rufer A</searchLink>; Roche Pharma Research and Early Development, Therapeutic Modalities, Lead Discovery, Roche Innovation Center Basel, F. Hoffmann-La Roche Ltd, Basel, Switzerland.<br /><searchLink fieldCode="AU" term="%22Heer+D%22">Heer D</searchLink>; Roche Pharma Research and Early Development, Therapeutic Modalities, Lead Discovery, Roche Innovation Center Basel, F. Hoffmann-La Roche Ltd, Basel, Switzerland.<br /><searchLink fieldCode="AU" term="%22Pfahler+N%22">Pfahler N</searchLink>; Roche Pharma Research and Early Development, Therapeutic Modalities, Lead Discovery, Roche Innovation Center Basel, F. 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Hoffmann-La Roche Ltd, Basel, Switzerland.<br /><searchLink fieldCode="AU" term="%22Fueth+M%22">Fueth M</searchLink>; Roche Pharma Research and Early Development, Pharmaceutical Science, Roche Innovation Center Basel, F. Hoffmann-La Roche Ltd, Basel, Switzerland.<br /><searchLink fieldCode="AU" term="%22Ebeling+M%22">Ebeling M</searchLink>; Roche Pharma Research and Early Development, Pharmaceutical Science, Roche Innovation Center Basel, F. Hoffmann-La Roche Ltd, Basel, Switzerland.<br /><searchLink fieldCode="AU" term="%22Villaseñor+R%22">Villaseñor R</searchLink>; 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.<br /><searchLink fieldCode="AU" term="%22Zhu+Y%22">Zhu Y</searchLink>; Department of Chemistry, Simon Fraser University, Burnaby, BC, V5A 1S6, Canada.<br /><searchLink fieldCode="AU" term="%22Deen+MC%22">Deen MC</searchLink>; Department of Chemistry, Simon Fraser University, Burnaby, BC, V5A 1S6, Canada.<br /><searchLink fieldCode="AU" term="%22Shan+X%22">Shan X</searchLink>; Department of Chemistry, Simon Fraser University, Burnaby, BC, V5A 1S6, Canada.<br /><searchLink fieldCode="AU" term="%22Ehsaei+Z%22">Ehsaei Z</searchLink>; Department of Biomedicine, University of Basel, Basel, Switzerland.<br /><searchLink fieldCode="AU" term="%22Taylor+V%22">Taylor V</searchLink>; Department of Biomedicine, University of Basel, Basel, Switzerland.<br /><searchLink fieldCode="AU" term="%22Sidransky+E%22">Sidransky E</searchLink>; Molecular Neurogenetics Section, National Human Genome Research Institute, Bethesda, MD, USA.<br /><searchLink fieldCode="AU" term="%22Vocadlo+DJ%22">Vocadlo DJ</searchLink>; 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.<br /><searchLink fieldCode="AU" term="%22Freskgård+PO%22">Freskgård PO</searchLink>; 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.<br /><searchLink fieldCode="AU" term="%22Jagasia+R%22">Jagasia R</searchLink>; 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.
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  Data: <searchLink fieldCode="JN" term="%22101528555%22">Nature communications</searchLink> [Nat Commun] 2023 Apr 12; Vol. 14 (1), pp. 2057. <i>Date of Electronic Publication: </i>2023 Apr 12.
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