한빛사 논문
Insung Kang,1,2,‡,†Je Min Yoo,3,5,‡Donghoon Kim,4Juhee Kim,3Myung Keun Cho,3Seung-EunLee,1,2Dong Jin Kim,7Byung-Chul Lee,1,2JinYoung Lee,1,2Jae-Jun Kim,1,2Nari Shin,1,2SoonWon Choi,1,2Young-Ho Lee,8,9,10Han Seok Ko,6Seokmin Shin,3Byung Hee Hong,3,4,7*andKyung-Sun Kang1,2*
1Adult Stem Cell Research Center, College of Veterinary Medicine, Seoul National University, Seoul 08826, Korea
2Research Institute for Veterinary Science, College of Veterinary Medicine, Seoul National University, Seoul 08826, Korea
3Department of Chemistry, Seoul National University, Seoul 08826, Korea
4BioGraphene Inc., Advanced Institute of Convergence Technology, Suwon 16229, Korea
5BioGraphene Inc.,555 West Fifth Street, Los Angeles, California 90013, United States.
6Department of Neurology & Neuroregeneration and Stem Cell Programs, Institute for Cell Engineering, The Johns Hopkins University School of Medicine, Baltimore, MD 21205, USA
7Graphene Square Inc. & Graphene Research Center, Advanced Institute of Convergence Technology, Suwon 16229, Korea
8Research Center for Bioconvergence Analysis, Korea Basic Science Institute, Chungcheongbuk-do 28119, Korea
9Bio-Analytical Science, University of Science and Technology, Daejeon 34113, Korea
10Graduate School of Analytical Science and Technology, Chungnam National University, Daejeon 34134, Korea
‡,*These authors contributed equally to this work.
†Present Address: Program in Developmental Endocrinology and Genetics, Eunice Kennedy Shriver National Institute of Child Health and Human Development, NIH, Bethesda, Maryland 20892, United State
Corresponding Authors: Byung Hee Hong and Kyung-Sun Kang
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.
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