Elevated tripeptidyl-peptidase 1 corrects multiple disease phenotypes in a mouse model of juvenile neuronal ceroid lipofuscinosis Article Swipe
Whitney Banach‐Petrosky
,
Katherine E. Larrimore
,
Elizabeth H Sleat
,
Allyson Bazer
,
Benjamin A. Samuels
,
Yixin Tan
,
Andrew C. Melton
,
Justin K. Ichida
,
Todd P. Logan
,
Peter Lobel
,
David E. Sleat
·
YOU?
·
· 2025
· Open Access
·
· DOI: https://doi.org/10.1016/j.omtm.2025.101587
YOU?
·
· 2025
· Open Access
·
· DOI: https://doi.org/10.1016/j.omtm.2025.101587
Juvenile neuronal ceroid lipofuscinosis (JNCL) is a neurodegenerative lysosomal storage disease caused by the loss of the endolysosomal transmembrane protein, CLN3. The main protein component of lysosomal storage material in JNCL is subunit c of mitochondrial ATP synthase (SCMAS), which is normally degraded within the lysosome by tripeptidyl-peptidase 1 (TPP1) during mitophagy. Previous studies have shown that TPP1 expression is elevated in JNCL, a potential compensatory response, while reduced levels of TPP1 exacerbate disease in a JNCL mouse model. These observations suggest a role for TPP1 in JNCL pathogenesis, and it is possible that lysosomal perturbations from the loss of CLN3 in JNCL could reduce the ability of TPP1 to degrade SCMAS. To test this hypothesis, we introduced a transgene that overexpresses TPP1 in a mouse model of JNCL and find that constitutively elevated TPP1 prevents SCMAS storage. This is associated with correction or significant reduction of other phenotypes of disease including neuroinflammation, an elevated plasma biomarker of neurodegeneration, and a disease-associated loss of brain mass with aging. From a clinical perspective, these results suggest that TPP1 augmentation could be a viable therapeutic strategy for JNCL and other lysosomal diseases that accumulate SCMAS where addressing the primary defect may be difficult or impossible.
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- Type
- article
- Language
- en
- Landing Page
- https://doi.org/10.1016/j.omtm.2025.101587
- OA Status
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- Related Works
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- OpenAlex ID
- https://openalex.org/W4413950966
All OpenAlex metadata
Raw OpenAlex JSON
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https://openalex.org/W4413950966Canonical identifier for this work in OpenAlex
- DOI
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https://doi.org/10.1016/j.omtm.2025.101587Digital Object Identifier
- Title
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Elevated tripeptidyl-peptidase 1 corrects multiple disease phenotypes in a mouse model of juvenile neuronal ceroid lipofuscinosisWork title
- Type
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articleOpenAlex work type
- Language
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enPrimary language
- Publication year
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2025Year of publication
- Publication date
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2025-09-03Full publication date if available
- Authors
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Whitney Banach‐Petrosky, Katherine E. Larrimore, Elizabeth H Sleat, Allyson Bazer, Benjamin A. Samuels, Yixin Tan, Andrew C. Melton, Justin K. Ichida, Todd P. Logan, Peter Lobel, David E. SleatList of authors in order
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https://doi.org/10.1016/j.omtm.2025.101587Publisher landing page
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YesWhether a free full text is available
- OA status
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goldOpen access status per OpenAlex
- OA URL
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https://doi.org/10.1016/j.omtm.2025.101587Direct OA link when available
- Concepts
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Neuronal ceroid lipofuscinosis, Phenotype, Batten disease, Juvenile, Lysosomal storage disease, Biology, Disease, Genetics, Medicine, Internal medicine, GeneTop concepts (fields/topics) attached by OpenAlex
- Cited by
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0Total citation count in OpenAlex
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10Other works algorithmically related by OpenAlex
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