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

Insung Kang, Je Min Yoo, Donghoon Kim, Juhee Kim, Myung Keun Cho, Seung Eun Lee, Dong Jin Kim, Byung Chul Lee, Jin Young Lee, Jae Jun Kim, Nari Shin, Soon Won Choi, Young Ho Lee, Han Seok Ko, Seokmin Shin, Byung Hee Hong, Kyung Sun Kang

Research output: Contribution to journalArticlepeer-review


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.

Original languageEnglish (US)
Pages (from-to)2339-2346
Number of pages8
JournalNano Letters
Issue number5
StatePublished - Mar 10 2021


  • Niemann-pick disease type C
  • cholesterol accumulation
  • graphene quantum dots
  • lysosomal storage disorders
  • nanomedicine

ASJC Scopus subject areas

  • Bioengineering
  • Chemistry(all)
  • Materials Science(all)
  • Condensed Matter Physics
  • Mechanical Engineering


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