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

Enzyme substitute remedy (ERT) can positively have an effect on the visceral manifestations of lysosomal storage illnesses (LSDs). However, the exclusion of the intravenous ERT brokers from the central nervous system (CNS) prevents direct therapeutic results. Using a neuronopathic Gaucher disease (nGD) mouse mannequin, CNS-ERT was created utilizing a systemic, non-invasive, and CNS-selective delivery system …

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