Target intelligence / Profile preview

Terminal galactose-containing glycans (Gal-glycans)

Target
Gal-glycans
Molecular classification
Glycan, Carbohydrate, Receptor
01

Overview

Cell-surface glycans, specifically those terminating in galactose residues on N-linked structures, serve as the primary attachment receptors for the adeno-associated virus rhesus isolate 10 (AAVrh10) [1][2]. These carbohydrate moieties are ubiquitously expressed across various tissues, including the central nervous system and liver, which facilitates the broad and potent transduction profile characteristic of AAVrh10-based vectors [2][3]. The viral entry process begins with the AAVrh10 capsid binding to these terminal galactose residues, a step that precedes the engagement of the universal proteinaceous co-receptor, Adeno-Associated Virus Receptor (AAVR/KIAA0319L), which mediates endocytosis [4]. In the field of gene therapy, these glycans are critical for the delivery of therapeutic transgenes, making AAVrh10 a prominent vector choice for treating neurodegenerative and metabolic disorders such as Alzheimer's disease and Mucopolysaccharidosis [3][5]. Therapeutic agents interacting with these glycans include various AAVrh10-delivered gene therapies currently in clinical development, such as LX1001 and RGX-121 [5][6]. Understanding the distribution and density of these cell-surface glycans is essential for optimizing vector design, predicting clinical efficacy, and managing potential off-target effects [1][2].

Other names
Terminal galactoseN-linked galactoseAAVrh10 receptor glycansGalactose residues
02

Mechanism of action

Primary attachment receptor for viral capsid binding and entry

03

Biological functions

Viral attachmentCellular entryProtein glycosylationCell-cell interaction
04

Disease associations

InfectionGenetic disordersNeurodegenerative diseaseLysosomal storage disease
05

Safety considerations

Off-target liver transductionPre-existing neutralizing antibodiesInnate immune response to viral capsidDose-dependent hepatotoxicity
06

Interacting drugs

LX1001

4 more in the full profile.

07

Biomarkers

Terminal galactose expression levelsAAVrh10 neutralizing antibody titerLectin binding (e.g., ECA lectin)

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