Target intelligence / Profile preview

Polycystic Kidney Disease 1

Molecular classification
Integral membrane protein, Receptor
01

Overview

The PKD1 gene encodes polycystin-1, a large integral membrane protein involved in regulating cell growth, division, migration, and intercellular interactions. It plays a critical role in sensing fluid flow within renal tubules and maintaining intracellular calcium homeostasis. Mutations in PKD1 cause autosomal dominant polycystic kidney disease (ADPKD).

Other names
PBPPC-1TRPP1
02

Mechanism of action

Modulation of polycystin-1 and polycystin-2 interaction

03

Biological functions

Regulation of cell growthCell division (proliferation)Cell migrationIntercellular interactionsFluid flow sensingCalcium homeostasisSignal transduction
04

Disease associations

Autosomal Dominant Polycystic Kidney Disease (ADPKD)Chronic Kidney DiseaseTumor suppressor (potential)
05

Safety considerations

Potential for off-target effects due to broad expressionChallenges in selectively targeting renal epithelial cells

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