Target intelligence / Profile preview

Cathepsin O (CTSO)

Target
CTSO
Molecular classification
Enzyme, Cysteine protease, Papain-like cathepsin family
01

Overview

Cathepsin O (CTSO) is a cysteine protease encoded by the CTSO gene, classified in the papain-like cathepsin family of enzymes. It is primarily involved in cellular protein degradation and turnover, influencing cell regulation and homeostasis. CTSO plays critical roles in cancer biology, notably by reducing protein levels of key regulatory proteins such as BRCA1 and ZNF423, thereby affecting tamoxifen response and serving as a prognostic marker in hormone receptor–positive breast cancer. Additionally, rare genetic variants in CTSO are linked to congenital developmental syndromes, and its expression is implicated in immune response and cellular defense. Pharmacologically, broad-spectrum cysteine protease inhibitors abolish CTSO activity, but no approved drugs selectively target CTSO. Potential therapeutic targeting is challenged by its fundamental role in normal cell processes and the risk of disrupting cellular homeostasis

Other names
Cathepsin OCTSOCTSO1CATO_HUMAN
02

Mechanism of action

Inhibition of cysteine protease activity. Reduction of protein degradation or turnover. Indirect modulation of signaling pathways critical to tumor progression.

03

Biological functions

Protein degradationProtein turnoverRegulation of cellular protein levelsModulation of tumor signaling pathwaysImmune responseCellular homeostasisCell death (via lysosomal membrane permeabilization)
04

Disease associations

Cancer (prognostic marker and modulates tamoxifen sensitivity via BRCA1 and ZNF423 regulation in breast cancer)Developmental disorders (rare variants found in congenital heterotaxy syndromes)Orofaciodigital syndrome VCone-rod dystrophy 1
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Safety considerations

Broad inhibition of cathepsins may disrupt normal protein turnover, cellular homeostasis, and immune response, leading to potential off-target effectsLMP (lysosomal membrane permeabilization) by cathepsins can contribute to cell death and may trigger unwanted apoptosis if dysregulated
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Interacting drugs

No approved drugs specifically target cathepsin O.

1 more in the full profile.

07

Biomarkers

CTSO protein levels can serve as a prognostic biomarker in hormone receptor–positive breast cancer, associated with tamoxifen sensitivity

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