Nanoreporter Identifies Lysosomal Storage Disease Lipid Accumulation Intracranially

Merav Antman-Passig, Zvi Yaari, Dana Goerzen, Rooshi Parikh, Savannah Chatman, Lauren E. Komer, Chen Chen, Emma Grabarnik, Mickael Mathieu, Adriana Haimovitz-Friedman, Daniel A. Heller*

*Corresponding author for this work

Research output: Contribution to journalArticlepeer-review

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.

Original languageAmerican English
Pages (from-to)10687-10695
Number of pages9
JournalNano Letters
Volume23
Issue number23
DOIs
StatePublished - 13 Dec 2023
Externally publishedYes

Bibliographical note

Publisher Copyright:
© 2023 American Chemical Society.

Keywords

  • Niemann−Pick
  • biosensor
  • carbon nanomaterials
  • neurodegenerative disease
  • sphingomyelin

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