Lack of cathepsin B increases severity in Niemann-Pick disease, type C1 mice.

Rachel Luke, Niamh X Cawley, Forbes D Porter

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Article in Neuroscience letters, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Not yet cited in PubMed.

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5 · Who and what money

Authors and funding

3 authors.

Rachel LukeSection on Molecular Dysmorphology, Division of Translational Medicine, Eunice Kennedy Shriver National Institute of Child Health and Human Development, National Institutes of Health, Bethesda, MD, USA.

Niamh X CawleySection on Molecular Dysmorphology, Division of Translational Medicine, Eunice Kennedy Shriver National Institute of Child Health and Human Development, National Institutes of Health, Bethesda, MD, USA.

Forbes D PorterSection on Molecular Dysmorphology, Division of Translational Medicine, Eunice Kennedy Shriver National Institute of Child Health and Human Development, National Institutes of Health, Bethesda, MD, USA. Electronic address: fdporter@mail.nih.gov.

Funding

Basic and Translational Studies of Inborn Errors of MetabolismZIAHD008988 · NICHD · EUNICE KENNEDY SHRIVER NATIONAL INSTITUTE OF CHILD HEALTH & HUMAN DEVELOPMENT · PI PORTER, FORBES · 2020 to 2025

$12.7M

Intramural NIH HHS ZIA HD008988

6 · The paper itself

Abstract

Neurodegeneration is a hallmark of Niemann-Pick disease type C1 (NPC1). One component in the pathology of neurodegeneration is increased lysosomal membrane permeability, especially in microglia. Leakage of lysosomal proteases, such as cathepsin B, into the cytoplasm and ultimately into the interstitial space can lead to activation of apoptosis and neuroinflammation. In line with previous reports showing the involvement of cathepsin B in neurodegeneration and the benefit of inhibiting cathepsin B to prevent neurodegeneration, we tested whether disease severity would be reduced in NPC1 mutant mice devoid of cathepsin B. Contrary to our expectation, the double mutant mice exhibited a more severe disease phenotype as measured by disease severity phenotypic scoring, beam walk latency test and a reduction in life expectancy. This highlights the importance of cathepsin B during postnatal development in the context of NPC1.

Indexed as

ApoptosisCathepsin BLysosome membrane permeabilityNeurodegenerationNPC1

Identifiers

PMID42668108

DOI10.1016/j.neulet.2026.138716

PMCPMC13540384

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