Target intelligence / Profile preview

Adeno-associated virus receptor (KIAA0319L) (AAVR)

Target
AAVR
Molecular classification
Receptor, Single-pass type I membrane protein, Glycoprotein
01

Overview

The Adeno-associated virus receptor (AAVR), encoded by the KIAA0319L gene, is a critical host cell surface protein required for the infection of most adeno-associated virus (AAV) serotypes, including AAV1, AAV2, AAV5, AAV8, and AAV9. It is a transmembrane glycoprotein that functions as a universal entry factor by binding to the AAV capsid and facilitating its trafficking from the plasma membrane through the endosomal system to the trans-Golgi network. While its endogenous biological role is linked to cell adhesion and potentially neuronal migration, its primary significance in biotechnology is as the essential gateway for AAV-based gene therapy vectors. The interaction between the AAV capsid and specific domains (PKD domains) of AAVR determines the efficiency of viral entry and subsequent transgene expression. Understanding AAVR is vital for optimizing gene delivery, as its expression levels across different tissues can influence the tropism and therapeutic efficacy of AAV-delivered genetic medicines.

Other names
KIAA0319-like proteinAAV receptorKIAA0319LAAV-RDyslexia-associated protein KIAA0319-like
02

Mechanism of action

The receptor facilitates the binding and internalization of AAV capsids into the host cell via receptor-mediated endocytosis and subsequent trafficking from the cell surface to the Golgi apparatus.

03

Biological functions

Viral entryEndosomal traffickingCell adhesionProtein binding
04

Disease associations

InfectionDyslexia (putative association)Genetic disease (as a vector target)
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Safety considerations

Variable transduction efficiency due to differential receptor expressionPotential for off-target tissue transductionPre-existing immunity to AAV capsids interfering with receptor binding
06

Interacting drugs

Adeno-associated virus vectors (e.g., Voretigene neparvovec, Onasemnogene abeparvovec)
07

Biomarkers

AAVR expression levelsKIAA0319L mRNA expression

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