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Author Notes:

Gang Wang, Email: wgneuron@hotmail.com

Ru-Jing Ren, Email: doctorren2001@126.com

QH, Y-BH, Y-FZ, R-JR, and GW designed the study. Y-BH, X-YX, RT, and R-JR performed the whole-genome sequencing-related work and clinical diagnosis. QH, Y-BH, Y-FZ, L-JL J-PL, J-TW, and X-QC performed the experiments and collected the data. QH, Y-BH, Y-FZ, ED, and GW analyzed the data. QH and GW wrote the manuscript. All authors contributed to the article and approved the submitted version.

We would like to thank the support from patient and her family for the innovation of modern science, Bio-Electron Microscopy Facility of Shanghai Tech University for their cryo-EM technical support, guidance on data visualization from Song Qingxiang in the Department of Pharmacology and Chemical Biology, Shanghai Jiao Tong University School of Medicine, and Quan Wang at the iHuman Institute, Shanghai Tech University for his critical reading.

The authors declare that the research was conducted in the absence of any commercial or financial relationships that could be construed as a potential conflict of interest.

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Research Funding:

This work was supported by grants from the National Natural Science Foundation of China (81971068 and 81801045), Natural Science Foundation of Shanghai (19ZR1431500), and Shanghai “Rising Stars of Medical Talent” Youth Development Program-Outstanding Youth Medical Talents (2018).

Keywords:

  • Science & Technology
  • Life Sciences & Biomedicine
  • Geriatrics & Gerontology
  • Neurosciences
  • Neurosciences & Neurology
  • cysteine string protein alpha
  • adult-onset neuronal ceroid lipofuscinosis
  • neurodegenerative disease
  • lysosome
  • cognitive decline
  • CSP-ALPHA
  • CYSTEINE
  • AGGREGATION
  • BINDING

Adult-Onset Neuronal Ceroid Lipofuscinosis With a Novel DNAJC5 Mutation Exhibits Aberrant Protein Palmitoylation

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Journal Title:

FRONTIERS IN AGING NEUROSCIENCE

Volume:

Volume 14

Publisher:

, Pages 829573-829573

Type of Work:

Article | Final Publisher PDF

Abstract:

Neuronal ceroid lipofuscinosis (NCL) is composed of a group of inherited neurodegenerative diseases, with the hallmark of lipofuscin deposit (a mixture of lipids and proteins with metal materials) inside the lysosomal lumen, which typically emits auto-fluorescence. Adult-onset NCL (ANCL) has been reported to be associated with a mutation in the DNAJC5 gene, including L115R, L116Δ, and the recently identified C124_C133dup mutation. In this study, we reported a novel C128Y mutation in a young Chinese female with ANCL, and this novel mutation caused abnormal palmitoylation and triggered lipofuscin deposits.

Copyright information:

© 2022 Huang, Zhang, Li, Dammer, Hu, Xie, Tang, Li, Wang, Che, Wang and Ren.

This is an Open Access work distributed under the terms of the Creative Commons Attribution 4.0 International License (https://creativecommons.org/licenses/by/4.0/rdf).
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