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Graphene Quantum Dots Alleviate Impaired Functions in Niemann-Pick Disease Type C in Vivo

Cited 7 time in Web of Science Cited 7 time in Scopus
Issue Date
2021-03
Citation
Nano Letters, Vol.21 No.5, pp.2339-2346
Keywords
graphene quantum dots;nanomedicine;lysosomal storage disorders;Niemann-pick disease type C;cholesterol accumulation
Abstract
While the neuropathological characteristics of Niemann-Pick disease type C (NPC) result in a fatal diagnosis, the development of clinically available therapeutic agent remains a challenge. Here we propose graphene quantum dots (GQDs) as a potential candidate for the impaired functions in NPC in vivo. In addition to the previous findings that GQDs exhibit negligible long-term toxicity and are capable of penetrating the blood-brain barrier, GQD treatment reduces the aggregation of cholesterol in the lysosome through expressed physical interactions. GQDs also promote autophagy and restore defective autophagic flux, which, in turn, decreases the atypical accumulation of autophagic vacuoles. More importantly, the injection of GQDs inhibits the loss of Purkinje cells in the cerebellum while also demonstrating reduced activation of microglia. The ability of GQDs to alleviate impaired functions in NPC proves the promise and potential of the use of GQDs toward resolving NPC and other related disorders.
ISSN
1530-6984
URI
https://hdl.handle.net/10371/178080
DOI
https://doi.org/10.1021/acs.nanolett.0c03741
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