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

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Graphene Quantum Dots Alleviate Impaired Functions in Niemann-Pick Disease Type C in Vivo
Nano Letters ( IF 9.6 ) Pub Date : 2021-01-20 , DOI: 10.1021/acs.nanolett.0c03741
Insung Kang _{1,

2} , Je Min Yoo _{3,

4} , Donghoon Kim ₅ , Juhee Kim ₃ , Myung Keun Cho ₃ , Seung-Eun Lee _{1,

2} , Dong Jin Kim ₆ , Byung-Chul Lee _{1,

2} , Jin Young Lee _{1,

2} , Jae-Jun Kim _{1,

2} , Nari Shin _{1,

2} , Soon Won Choi _{1,

2} , Young-Ho Lee _{7,

8,

9} , Han Seok Ko ₁₀ , Seokmin Shin ₃ , Byung Hee Hong _{3,

5,

6} , Kyung-Sun Kang _{1,

2}

Affiliation

Adult Stem Cell Research Center, College of Veterinary Medicine, Seoul National University, Seoul 08826, Korea.
Research Institute for Veterinary Science, College of Veterinary Medicine, Seoul National University, Seoul 08826, Korea.
Department of Chemistry, College of Natural Sciences, Seoul National University, Seoul 08826, Korea.
BioGraphene Inc., 555 West Fifth Street, Los Angeles, California 90013, United States.
BioGraphene Inc., Advanced Institute of Convergence Technology, Suwon 16229, Korea.
Graphene Square Inc. & Graphene Research Center, Advanced Institute of Convergence Technology, Suwon 16229, Korea.
Research Center for Bioconvergence Analysis, Korea Basic Science Institute, Chungcheongbuk-do 28119, Korea.
Bio-Analytical Science, University of Science and Technology, Daejeon 34113, Korea.
Graduate School of Analytical Science and Technology, Chungnam National University, Daejeon 34134, Korea.
Department of Neurology & Neuroregeneration and Stem Cell Programs, Institute for Cell Engineering, The Johns Hopkins University School of Medicine, Baltimore, Maryland 21205, United States.

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.

中文翻译：

石墨烯量子点减轻体内C型尼曼-皮克病的功能受损

虽然C型Niemann-Pick疾病（NPC）的神经病理学特征导致致命的诊断，但临床上可利用的治疗剂的开发仍然是一个挑战。在这里，我们提出石墨烯量子点（GQDs）作为体内NPC功能受损的潜在候选者。除了先前的发现，GQDs表现出可忽略的长期毒性并且能够穿透血脑屏障外，GQD治疗还通过表达的物理相互作用减少了溶酶体中胆固醇的聚集。GQD还促进自噬并恢复有缺陷的自噬通量，从而减少自噬泡的非典型积累。更重要的是，注射GQD抑制了小脑浦肯野细胞的损失，同时还证明了小胶质细胞的激活减少。

更新日期：2021-03-10

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