Nanoreporter identifies lysosomal storage disease lipid accumulation intracranially Journal Article


Authors: Antman-Passig, M.; Yaari, Z.; Goerzen, D.; Parikh, R.; Chatman, S.; Komer, L. E.; Chen, C.; Grabarnik, E.; Mathieu, M.; Haimovitz-Friedman, A.; Heller, D. A.
Article Title: Nanoreporter identifies lysosomal storage disease lipid accumulation intracranially
Abstract: Dysregulated lipid metabolism contributes to neurodegenerative pathologies and neurological decline in lysosomal storage disorders as well as more common neurodegenerative diseases. Niemann-Pick type A (NPA) is a fatal neurodegenerative lysosomal storage disease characterized by abnormal sphingomyelin accumulation in the endolysosomal lumen. The ability to monitor abnormalities in lipid homeostasis intracranially could improve basic investigations and the development of effective treatment strategies. We investigated the carbon nanotube-based detection of intracranial lipid content. We found that the near-infrared emission of a carbon nanotube-based lipid sensor responds to lipid accumulation in neuronal and in vivo models of NPA. The nanosensor detected lipid accumulation intracranially in an acid sphingomyelinase knockout mouse via noninvasive near-infrared spectroscopy. This work indicates a tool to improve drug development processes in NPA, other lysosomal storage diseases, and neurodegenerative diseases. © 2023 American Chemical Society.
Keywords: mouse; animal; metabolism; animals; mice; neurons; pathology; nerve cell; degenerative disease; lysosome; lysosome storage disease; lysosomes; phospholipids; neurodegenerative diseases; sphingomyelin; infrared devices; carbon nanotube; nanotubes, carbon; carbon nanotubes; lipid homeostasis; near infrared spectroscopy; sphingomyelins; lysosomal storage diseases; biosensor; neurodegenerative disease; neurodegenerative; near-infrared emissions; lysosomal storage disorder; lipid metabolisms; carbon nanomaterials; niemann−pick; carbon nano-materials; lipid accumulations; lipid content
Journal Title: Nano Letters
Volume: 23
Issue: 23
ISSN: 1530-6984
Publisher: American Chemical Society  
Date Published: 2023-12-13
Start Page: 10687
End Page: 10695
Language: English
DOI: 10.1021/acs.nanolett.3c02502
PUBMED: 37889874
PROVIDER: scopus
PMCID: PMC11246544
DOI/URL:
Notes: Article -- MSK Cancer Center Support Grant (P30 CA008748) acknowledged in PDF -- MSK corresponding author is Daniel Heller -- Source: Scopus
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