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  1. Article ; Online: Extracellular Vesicles Released by Genetically Modified Macrophages Activate Autophagy and Produce Potent Neuroprotection in Mouse Model of Lysosomal Storage Disorder, Batten Disease.

    El-Hage, Nazira / Haney, Matthew J / Zhao, Yuling / Rodriguez, Myosotys / Wu, Zhanhong / Liu, Mori / Swain, Carson J / Yuan, Hong / Batrakova, Elena V

    Cells

    2023  Volume 12, Issue 11

    Abstract: Over the recent decades, the use of extracellular vesicles (EVs) has attracted considerable attention. Herein, we report the development of a novel EV-based drug delivery system for the transport of the lysosomal enzyme tripeptidyl peptidase-1 (TPP1) to ... ...

    Abstract Over the recent decades, the use of extracellular vesicles (EVs) has attracted considerable attention. Herein, we report the development of a novel EV-based drug delivery system for the transport of the lysosomal enzyme tripeptidyl peptidase-1 (TPP1) to treat Batten disease (BD). Endogenous loading of macrophage-derived EVs was achieved through transfection of parent cells with TPP1-encoding
    MeSH term(s) Mice ; Animals ; Neuronal Ceroid-Lipofuscinoses/metabolism ; Serine Proteases/genetics ; Aminopeptidases/genetics ; Dipeptidyl-Peptidases and Tripeptidyl-Peptidases/metabolism ; Lipofuscin/metabolism ; Lipofuscin/therapeutic use ; Neuroprotection ; Tripeptidyl-Peptidase 1 ; Lysosomal Storage Diseases/metabolism ; Extracellular Vesicles/metabolism ; Lysosomes/metabolism ; Autophagy
    Chemical Substances Serine Proteases (EC 3.4.-) ; Aminopeptidases (EC 3.4.11.-) ; Dipeptidyl-Peptidases and Tripeptidyl-Peptidases (EC 3.4.14.-) ; Lipofuscin ; Tripeptidyl-Peptidase 1
    Language English
    Publishing date 2023-05-29
    Publishing country Switzerland
    Document type Journal Article ; Research Support, N.I.H., Extramural ; Research Support, Non-U.S. Gov't
    ZDB-ID 2661518-6
    ISSN 2073-4409 ; 2073-4409
    ISSN (online) 2073-4409
    ISSN 2073-4409
    DOI 10.3390/cells12111497
    Database MEDical Literature Analysis and Retrieval System OnLINE

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  2. Article ; Online: Using Extracellular Vesicles Released by GDNF-Transfected Macrophages for Therapy of Parkinson Disease.

    Zhao, Yuling / Haney, Matthew J / Fallon, John K / Rodriguez, Myosotys / Swain, Carson J / Arzt, Camryn J / Smith, Philip C / Loop, Matthew Shane / Harrison, Emily B / El-Hage, Nazira / Batrakova, Elena V

    Cells

    2022  Volume 11, Issue 12

    Abstract: Extracellular vesicles (EVs) are cell-derived nanoparticles that facilitate transport of proteins, lipids, and genetic material, playing important roles in intracellular communication. They have remarkable potential as non-toxic and non-immunogenic ... ...

    Abstract Extracellular vesicles (EVs) are cell-derived nanoparticles that facilitate transport of proteins, lipids, and genetic material, playing important roles in intracellular communication. They have remarkable potential as non-toxic and non-immunogenic nanocarriers for drug delivery to unreachable organs and tissues, in particular, the central nervous system (CNS). Herein, we developed a novel platform based on macrophage-derived EVs to treat Parkinson disease (PD). Specifically, we evaluated the therapeutic potential of EVs secreted by autologous macrophages that were transfected ex vivo to express glial-cell-line-derived neurotrophic factor (GDNF). EV-GDNF were collected from conditioned media of GDNF-transfected macrophages and characterized for GDNF content, size, charge, and expression of EV-specific proteins. The data revealed that, along with the encoded neurotrophic factor, EVs released by pre-transfected macrophages carry GDNF-encoding DNA. Four-month-old transgenic Parkin Q311(X)A mice were treated with EV-GDNF via intranasal administration, and the effect of this therapeutic intervention on locomotor functions was assessed over a year. Significant improvements in mobility, increases in neuronal survival, and decreases in neuroinflammation were found in PD mice treated with EV-GDNF. No offsite toxicity caused by EV-GDNF administration was detected. Overall, an EV-based approach can provide a versatile and potent therapeutic intervention for PD.
    MeSH term(s) Animals ; Central Nervous System ; Extracellular Vesicles/metabolism ; Glial Cell Line-Derived Neurotrophic Factor/genetics ; Glial Cell Line-Derived Neurotrophic Factor/metabolism ; Macrophages/metabolism ; Mice ; Parkinson Disease/genetics ; Parkinson Disease/metabolism ; Parkinson Disease/therapy
    Chemical Substances Glial Cell Line-Derived Neurotrophic Factor
    Language English
    Publishing date 2022-06-15
    Publishing country Switzerland
    Document type Journal Article ; Research Support, Non-U.S. Gov't ; Research Support, N.I.H., Extramural
    ZDB-ID 2661518-6
    ISSN 2073-4409 ; 2073-4409
    ISSN (online) 2073-4409
    ISSN 2073-4409
    DOI 10.3390/cells11121933
    Database MEDical Literature Analysis and Retrieval System OnLINE

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    Kategorien

    Order via subito

    This service is chargeable due to the Delivery terms set by subito. Orders including an article and supplementary material will be classified as separate orders. In these cases, fees will be demanded for each order.

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