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Polymeric nanoparticle-mediated GBA1 gene therapy is neuroprotective in a preclinical model of Parkinson's disease.

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Title: Polymeric nanoparticle-mediated GBA1 gene therapy is neuroprotective in a preclinical model of Parkinson's disease.
Authors: Kwatra M; Institute for Cell Engineering, School of Medicine, Johns Hopkins University, Baltimore, MD, 21205, USA.; Department of Neurology, School of Medicine, Johns Hopkins University, Baltimore, MD, 21205, USA.; Department of Ophthalmology, College of Medicine, Medical University of South Carolina (MUSC), Charleston, SC, 29425, USA., Kwak G; Department of Neurosurgery, School of Medicine, University of Maryland, Baltimore, MD, 21201, USA.; Medicine Institute for Neuroscience Discovery (UM-MIND), School of Medicine, University of Maryland, Baltimore, MD, 21201, USA., Li H; Department of Neurosurgery, School of Medicine, University of Maryland, Baltimore, MD, 21201, USA.; Medicine Institute for Neuroscience Discovery (UM-MIND), School of Medicine, University of Maryland, Baltimore, MD, 21201, USA.; Department of Chemical and Biomolecular Engineering, School of Engineering, Johns Hopkins University, Baltimore, MD, 21218, USA., Suk JS; Department of Neurosurgery, School of Medicine, University of Maryland, Baltimore, MD, 21201, USA. jsuk@som.umaryland.edu.; Medicine Institute for Neuroscience Discovery (UM-MIND), School of Medicine, University of Maryland, Baltimore, MD, 21201, USA. jsuk@som.umaryland.edu.; Department of Chemical and Biomolecular Engineering, School of Engineering, Johns Hopkins University, Baltimore, MD, 21218, USA. jsuk@som.umaryland.edu.; Department of Neurosurgery, School of Medicine, Johns Hopkins University, Baltimore, MD, 21205, USA. jsuk@som.umaryland.edu., Ko HS; Institute for Cell Engineering, School of Medicine, Johns Hopkins University, Baltimore, MD, 21205, USA. hko3@jhmi.edu.; Department of Neurology, School of Medicine, Johns Hopkins University, Baltimore, MD, 21205, USA. hko3@jhmi.edu.
Source: Drug delivery and translational research [Drug Deliv Transl Res] 2026 Mar; Vol. 16 (3), pp. 894-910. Date of Electronic Publication: 2025 Aug 20.
Publication Type: Journal Article
Journal Info: Publisher: Springer Country of Publication: United States NLM ID: 101540061 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 2190-3948 (Electronic) Linking ISSN: 2190393X NLM ISO Abbreviation: Drug Deliv Transl Res Subsets: MEDLINE
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  Data: Polymeric nanoparticle-mediated GBA1 gene therapy is neuroprotective in a preclinical model of Parkinson's disease.
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  Data: <searchLink fieldCode="AU" term="%22Kwatra+M%22">Kwatra M</searchLink>; Institute for Cell Engineering, School of Medicine, Johns Hopkins University, Baltimore, MD, 21205, USA.; Department of Neurology, School of Medicine, Johns Hopkins University, Baltimore, MD, 21205, USA.; Department of Ophthalmology, College of Medicine, Medical University of South Carolina (MUSC), Charleston, SC, 29425, USA.<br /><searchLink fieldCode="AU" term="%22Kwak+G%22">Kwak G</searchLink>; Department of Neurosurgery, School of Medicine, University of Maryland, Baltimore, MD, 21201, USA.; Medicine Institute for Neuroscience Discovery (UM-MIND), School of Medicine, University of Maryland, Baltimore, MD, 21201, USA.<br /><searchLink fieldCode="AU" term="%22Li+H%22">Li H</searchLink>; Department of Neurosurgery, School of Medicine, University of Maryland, Baltimore, MD, 21201, USA.; Medicine Institute for Neuroscience Discovery (UM-MIND), School of Medicine, University of Maryland, Baltimore, MD, 21201, USA.; Department of Chemical and Biomolecular Engineering, School of Engineering, Johns Hopkins University, Baltimore, MD, 21218, USA.<br /><searchLink fieldCode="AU" term="%22Suk+JS%22">Suk JS</searchLink>; Department of Neurosurgery, School of Medicine, University of Maryland, Baltimore, MD, 21201, USA. jsuk@som.umaryland.edu.; Medicine Institute for Neuroscience Discovery (UM-MIND), School of Medicine, University of Maryland, Baltimore, MD, 21201, USA. jsuk@som.umaryland.edu.; Department of Chemical and Biomolecular Engineering, School of Engineering, Johns Hopkins University, Baltimore, MD, 21218, USA. jsuk@som.umaryland.edu.; Department of Neurosurgery, School of Medicine, Johns Hopkins University, Baltimore, MD, 21205, USA. jsuk@som.umaryland.edu.<br /><searchLink fieldCode="AU" term="%22Ko+HS%22">Ko HS</searchLink>; Institute for Cell Engineering, School of Medicine, Johns Hopkins University, Baltimore, MD, 21205, USA. hko3@jhmi.edu.; Department of Neurology, School of Medicine, Johns Hopkins University, Baltimore, MD, 21205, USA. hko3@jhmi.edu.
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        Value: 10.1007/s13346-025-01944-3
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        Text: English
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              Text: 2026 Mar
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