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Targeting neuronal lysosomal dysfunction caused by ß-glucocerebrosidase deficiency with an enzyme-based brain shuttle construct.
Gehrlein, Alexandra; Udayar, Vinod; Anastasi, Nadia; Morella, Martino L; Ruf, Iris; Brugger, Doris; von der Mark, Sophia; Thoma, Ralf; Rufer, Arne; Heer, Dominik; Pfahler, Nina; Jochner, Anton; Niewoehner, Jens; Wolf, Luise; Fueth, Matthias; Ebeling, Martin; Villaseñor, Roberto; Zhu, Yanping; Deen, Matthew C; Shan, Xiaoyang; Ehsaei, Zahra; Taylor, Verdon; Sidransky, Ellen; Vocadlo, David J; Freskgård, Per-Ola; Jagasia, Ravi.
Afiliação
  • 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.
  • Ruf I; Department of Anatomy and Neurosciences, Amsterdam University Medical Center | VUmc, Amsterdam, Netherlands.
  • 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.
  • Jochner A; Roche Pharma Research and Early Development, Therapeutic Modalities, Lead Discovery, Roche Innovation Center Basel, F. Hoffmann-La Roche Ltd, Basel, Switzerland.
  • Niewoehner J; Interfaculty Institute of Biochemistry & Structural Biology Biochemistry (IFIB), Eberhard Karls University of Tübingen, Tübingen, Germany.
  • Wolf L; Roche Pharma Research and Early Development, Therapeutic Modalities Large Molecule Research, Roche Innovation Center Munich, Roche Diagnostics GmbH, Penzberg, Germany.
  • Fueth M; Roche Pharma Research and Early Development, Therapeutic Modalities Large Molecule Research, Roche Innovation Center Munich, Roche Diagnostics GmbH, Penzberg, Germany.
  • Ebeling M; Roche Pharma Research and Early Development, Data & Analytics, Roche Innovation Center Basel, F. Hoffmann-La Roche Ltd, Basel, Switzerland.
  • Villaseñor R; Roche Pharma Research and Early Development, Pharmaceutical Science, Roche Innovation Center Basel, F. Hoffmann-La Roche Ltd, Basel, Switzerland.
  • Zhu Y; Roche Pharma Research and Early Development, Pharmaceutical Science, Roche Innovation Center Basel, F. Hoffmann-La Roche Ltd, Basel, Switzerland.
  • Deen MC; 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.
  • Shan X; Department of Chemistry, Simon Fraser University, Burnaby, BC, V5A 1S6, Canada.
  • Ehsaei Z; Department of Chemistry, Simon Fraser University, Burnaby, BC, V5A 1S6, Canada.
  • Taylor V; Department of Chemistry, Simon Fraser University, Burnaby, BC, V5A 1S6, Canada.
  • Sidransky E; Department of Biomedicine, University of Basel, Basel, Switzerland.
  • Vocadlo DJ; Department of Biomedicine, University of Basel, Basel, Switzerland.
  • Freskgård PO; Molecular Neurogenetics Section, National Human Genome Research Institute, Bethesda, MD, USA.
  • Jagasia R; Department of Chemistry, Simon Fraser University, Burnaby, BC, V5A 1S6, Canada.
Nat Commun ; 14(1): 2057, 2023 04 12.
Article em En | MEDLINE | ID: mdl-37045813

Texto completo: 1 Bases de dados: MEDLINE Assunto principal: Doença de Parkinson / Doença de Gaucher Tipo de estudo: Prognostic_studies / Risk_factors_studies Limite: Animals Idioma: En Revista: Nat Commun Assunto da revista: BIOLOGIA / CIENCIA Ano de publicação: 2023 Tipo de documento: Article País de afiliação: Suíça

Texto completo: 1 Bases de dados: MEDLINE Assunto principal: Doença de Parkinson / Doença de Gaucher Tipo de estudo: Prognostic_studies / Risk_factors_studies Limite: Animals Idioma: En Revista: Nat Commun Assunto da revista: BIOLOGIA / CIENCIA Ano de publicação: 2023 Tipo de documento: Article País de afiliação: Suíça