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Systemic enzyme delivery by blood-brain barrier-penetrating SapC-DOPS nanovesicles for treatment of neuronopathic Gaucher disease.
Sun, Ying; Liou, Benjamin; Chu, Zhengtao; Fannin, Venette; Blackwood, Rachel; Peng, Yanyan; Grabowski, Gregory A; Davis, Harold W; Qi, Xiaoyang.
Affiliation
  • Sun Y; Division of Human Genetics, Cincinnati Children's Hospital Medical Center, Cincinnati, OH, USA; Department of Pediatrics, University of Cincinnati College of Medicine, Cincinnati, OH, USA. Electronic address: ying.sun@cchmc.org.
  • Liou B; Division of Human Genetics, Cincinnati Children's Hospital Medical Center, Cincinnati, OH, USA.
  • Chu Z; Division of Hematology/Oncology, Department of Internal Medicine, University of Cincinnati, College of Medicine, Cincinnati, OH, USA.
  • Fannin V; Division of Human Genetics, Cincinnati Children's Hospital Medical Center, Cincinnati, OH, USA.
  • Blackwood R; Division of Human Genetics, Cincinnati Children's Hospital Medical Center, Cincinnati, OH, USA.
  • Peng Y; Division of Human Genetics, Cincinnati Children's Hospital Medical Center, Cincinnati, OH, USA.
  • Grabowski GA; Division of Human Genetics, Cincinnati Children's Hospital Medical Center, Cincinnati, OH, USA.
  • Davis HW; Division of Hematology/Oncology, Department of Internal Medicine, University of Cincinnati, College of Medicine, Cincinnati, OH, USA.
  • Qi X; Division of Human Genetics, Cincinnati Children's Hospital Medical Center, Cincinnati, OH, USA; Department of Pediatrics, University of Cincinnati College of Medicine, Cincinnati, OH, USA; Division of Hematology/Oncology, Department of Internal Medicine, University of Cincinnati, College of Medicine
EBioMedicine ; 55: 102735, 2020 May.
Article in En | MEDLINE | ID: mdl-32279952
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Full text: 1 Collection: 01-internacional Database: MEDLINE Main subject: Phosphatidylserines / Blood-Brain Barrier / Drug Delivery Systems / Saposins / Gaucher Disease / Glucosylceramidase Type of study: Prognostic_studies Aspects: Implementation_research Limits: Animals / Female / Humans / Male Language: En Journal: EBioMedicine Year: 2020 Document type: Article
Fulltext
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Full text: 1 Collection: 01-internacional Database: MEDLINE Main subject: Phosphatidylserines / Blood-Brain Barrier / Drug Delivery Systems / Saposins / Gaucher Disease / Glucosylceramidase Type of study: Prognostic_studies Aspects: Implementation_research Limits: Animals / Female / Humans / Male Language: En Journal: EBioMedicine Year: 2020 Document type: Article