Target intelligence / Profile preview

Lactosylceramide (LacCer)

Target
LacCer
Molecular classification
Glycosphingolipid, Diglycosylceramide, Lipid raft component, Pattern recognition receptor, Other
01

Overview

Lactosylceramide is a diglycosylated glycosphingolipid composed of a ceramide backbone covalently linked to a lactose moiety (specifically, β-D-galactosyl-(1→4)-β-D-glucosylceramide)[1][2][3]. It is found in small amounts in most animal tissues, enriched in the microdomains (lipid rafts) of plasma membranes, where it serves structural, signaling, and pattern-recognition functions[1][2][5][6][7]. Biosynthetically, lactosylceramide is a crucial intermediate, being the precursor to most complex glycosphingolipids including gangliosides and sulfatides, and is synthesized by β-1,4-galactosyltransferases in the Golgi apparatus[1][5][6]. It mediates cell proliferation, adhesion, migration, angiogenesis, apoptosis, and immune recognition, and is implicated in cardiovascular disease, cancer, Fabry disease, inflammation, neurodegeneration, and host-pathogen interactions[2][5][6][7]. Accumulation or dysregulation of lactosylceramide is pathogenic in several disorders, making its metabolic pathways a therapeutic target for small-molecule inhibitors and other interventions[2][5][6][7].

Other names
Cytolipin HCDw17 antigenCD17ceramide lactosideceramide dihexosideCDHN-lignoceroyl-1-sphingosyl lactosidebeta-D-galactosyl-(1-4)-D-glucosylceramidebeta-D-galactosyl-1,4-beta-D-glucosylceramideglycosyl-N-acylsphingosine
02

Mechanism of action

Inhibition of synthesis (by targeting LacCer synthase, beta-1,4-galactosyltransferase); Modulation of downstream signaling pathways (e.g., inhibition of oxygen-sensitive signaling, modulation of immune cell responses)

03

Biological functions

Biosynthetic precursor of more complex glycosphingolipids (gangliosides, sulfatides, oligoglycosylceramides)Structural role in stabilizing plasma membrane and lipid raftsActivator of receptor molecules in microdomainsPattern recognition/binding to pathogenic bacteria (innate immune recognition)Participant in cell signaling, particularly oxygen-sensitive pathways related to proliferation, adhesion, migration, and angiogenesisInducer of apoptosis
04

Disease associations

CancerCardiovascular diseaseInflammation and inflammatory diseasesFabry diseaseNeurodegenerative diseaseInfection
05

Safety considerations

Targeting of glycosphingolipid metabolism can impact numerous essential pathways and may lead to off-target effects in neural and immune systemsAccumulation or depletion can have deleterious effects, as in storage diseases and cancer biology
06

Interacting drugs

None specifically approved as direct drugs; some investigative small-molecule and enzyme inhibitors of glycosphingolipid biosynthesis (e.g., eliglustat for related sphingolipidoses, but not direct to LacCer)
07

Biomarkers

Elevated tissue or plasma LacCer used as a biomarker in certain lysosomal storage diseases (e.g., Fabry disease), cardiovascular disease, and as a marker of immune activation and cancer progression

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