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Target intelligence / Profile preview
AAV receptor (AAVR)
- Target
- AAVR
- Molecular classification
- Receptor, Cell-surface protein
01
Overview
The AAV receptor (AAVR) is a cell-surface protein that serves as the primary point of entry for adeno-associated virus vectors in mammalian cells[2][5]. Engineering the interaction between AAV capsids and cell-surface receptors (AAVR or tissue-specific proteins such as integrins) enables selective and efficient gene delivery to neuromuscular tissue[4][3]. Strategies include overexpression of AAVR, capsid engineering to enhance receptor affinity, and development of AAV variants with tissue selectivity, which collectively improve transduction efficiency and the safety profile of gene therapy for diseases such as muscular dystrophies[3][4].
- Other names
- Adeno-associated virus receptorKIAA0319L
02
Mechanism of action
Enhanced tissue-specific gene transduction by engineered AAV capsids binding selectively to AAVR or muscle/tissue-enriched receptors
03
Biological functions
Mediates adeno-associated virus entry into cellsRegulates gene delivery efficiency and tissue specificity
04
Disease associations
Gene therapy for neuromuscular diseases, e.g., muscular dystrophiesOther roles depend on the engineered vector and tissue targeting strategy
05
Safety considerations
Immune response to viral capsid or transgene productOff-target transduction leading to ectopic gene expressionInflammatory responses in injected tissues, especially with certain AAV serotypes
06
Biomarkers
Expression level of AAVR in target tissue for gene therapy efficiency prediction
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