Target intelligence / Profile preview

Pigment epithelium-derived factor (PEDF) (PEDF)

Target
PEDF
Molecular classification
Serpin, Neurotrophic factor, Angiogenesis inhibitor, Glycoprotein
01

Overview

Pigment epithelium-derived factor (PEDF), encoded by the SERPINF1 gene, is a multifunctional secreted glycoprotein that serves as a critical regulator of the corneal limbal stem cell (LSC) niche. Although it is a member of the serpin superfamily, it lacks protease inhibitory activity and instead functions as one of the body's most potent anti-angiogenic and neurotrophic factors. In the ocular environment, PEDF-responsive pathways are essential for preserving the regenerative capacity of LSCs and maintaining the transparency of the cornea by preventing pathological blood vessel encroachment. PEDF exerts its biological effects by binding to specific receptors, most notably the phospholipase-linked PEDF-R (PNPLA2) and the Wnt-pathway co-receptor LRP6. Dysregulation of these pathways is a hallmark of limbal stem cell deficiency (LSCD) and various retinal degenerative diseases. Therapeutic strategies currently under investigation include the use of recombinant PEDF, synthetic PEDF-derived peptides, and gene therapy to restore ocular surface homeostasis and promote corneal wound healing. (Sources: UniProt P36955; PubMed: 23554555, 28205514).

Other names
Serpin F1SERPINF1EPC-1Early population doubling level cDNA 1Cell proliferation-inhibiting gene 35 protein
02

Mechanism of action

PEDF binds to the PEDF-R (PNPLA2) receptor to activate phospholipase activity and interacts with the Wnt co-receptor LRP6 to modulate downstream signaling pathways, thereby maintaining the stemness and quiescence of limbal stem cells while antagonizing VEGF-mediated angiogenesis.

03

Biological functions

Stem cell maintenanceAnti-angiogenesisNeuroprotectionCell proliferationSignal transductionAnti-inflammation
04

Disease associations

Limbal stem cell deficiencyCorneal neovascularizationAge-related macular degenerationDiabetic retinopathyCancer metastasis
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Safety considerations

Off-target effects on systemic lipid metabolism via PNPLA2/ATGLPotential for ocular fibrosis with chronic overexpressionStability and delivery challenges to the avascular cornea
06

Interacting drugs

Recombinant human PEDF (rhPEDF)

2 more in the full profile.

07

Biomarkers

PEDF protein levels in tearsp63 expressionABCB5 expressionCytokeratin 12 (K12)

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