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

doi:10.1021/acs.nanolett.3c02502

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 journal › Article › peer-review

7 Scopus citations

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 language	English
Pages (from-to)	10687-10695
Number of pages	9
Journal	Nano Letters
Volume	23
Issue number	23
DOIs	https://doi.org/10.1021/acs.nanolett.3c02502
State	Published - 13 Dec 2023
Externally published	Yes

Bibliographical note

Publisher Copyright:
© 2023 American Chemical Society.

Keywords

Niemann−Pick
biosensor
carbon nanomaterials
neurodegenerative disease
sphingomyelin

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10.1021/acs.nanolett.3c02502

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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.",

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

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doi = "10.1021/acs.nanolett.3c02502",

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AU - Antman-Passig, Merav

AU - Yaari, Zvi

AU - Goerzen, Dana

AU - Parikh, Rooshi

AU - Chatman, Savannah

AU - Komer, Lauren E.

AU - Chen, Chen

AU - Grabarnik, Emma

AU - Mathieu, Mickael

AU - Haimovitz-Friedman, Adriana

AU - Heller, Daniel A.

PY - 2023/12/13

Y1 - 2023/12/13

N2 - 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.

AB - 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.

KW - Niemann−Pick

KW - biosensor

KW - carbon nanomaterials

KW - neurodegenerative disease

KW - sphingomyelin

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Nanoreporter Identifies Lysosomal Storage Disease Lipid Accumulation Intracranially

Abstract

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Keywords

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