Target intelligence / Profile preview

Cellular Prion Protein (PrPC)

Target
PrPC
Molecular classification
Glycoprotein, GPI-anchored protein, Cell surface receptor
01

Overview

Cellular prion protein is an essential CNS-expressed GPI-linked glycoprotein involved primarily with metal ion homeostasis, neuroprotection, signal transduction scaffolding—and whose misfolded state underlies fatal transmissible neurodegenerative disorders. PrPC acts as a dynamic cell-surface scaffold protein capable of assembling signaling modules by interacting with various extracellular ligands. This enables modulation of intracellular signaling cascades relevant to neuronal survival and plasticity. Aberrant misfolding of PrPC into PrPSc is the hallmark of prion diseases.

Other names
Prion proteinPRNP
02

Mechanism of action

Modulation of PrPC misfolding, prevention of PrPSc formation, inhibition of PrPC-amyloid beta interaction (potential)

03

Biological functions

Copper homeostasisNeuroprotectionSignal transductionCell adhesionStem cell renewalMemory formation
04

Disease associations

Prion diseases (Creutzfeldt-Jakob disease, bovine spongiform encephalopathy, scrapie, chronic wasting disease)Alzheimer's disease
05

Safety considerations

Potential for off-target effects due to broad expressionBlood-brain barrier penetration challenges for therapeutic agents
06

Biomarkers

PrPC levels in cerebrospinal fluid (diagnostic/prognostic for prion diseases)PrPSc detection (diagnostic for prion diseases)

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