Systemic enzyme delivery by blood-brain barrier-penetrating SapC-DOPS nanovesicles for treatment of neuronopathic Gaucher disease.

Systemic enzyme delivery by blood-brain barrier-penetrating SapC-DOPS nanovesicles for treatment of neuronopathic Gaucher disease. EBioMedicine. 2020 May; 55:102735.

View in: PubMed

subject areas

Animals
Biological Transport
Blood-Brain Barrier
Disease Models, Animal
Drug Delivery Systems
Drug Stability
Enzyme Replacement Therapy
Female
Gaucher Disease
Glucosylceramidase
Humans
Male
Mice
Mice, Transgenic
Nanostructures
Permeability
Phosphatidylserines
Psychomotor Performance
Saposins
Survival Analysis
Treatment Outcome

authors with profiles

Gregory Grabowski