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Target intelligence / Profile preview
Adeno-Associated Virus (AAV) Vector (AAV)
- Target
- AAV
- Molecular classification
- Viral Vector, Gene Delivery System
01
Overview
Adeno-associated virus (AAV) is a small, non-enveloped virus from the parvovirus family that is used as a vector for gene delivery in gene therapy. Recombinant AAV vectors are engineered to deliver therapeutic genes to target tissues without causing disease. AAV vectors offer advantages such as non-pathogenicity, broad tropism, low integration rate, and customizability. However, they have limitations, including limited packaging capacity and potential pre-existing immunity. AAV-mediated gene delivery has been approved for treating various genetic diseases.
- Other names
- rAAVRecombinant Adeno-Associated VirusAAV Gene Therapy Vector
02
Mechanism of action
AAV vectors deliver therapeutic genes to target cells. The vector enters the cell via receptor-mediated endocytosis, translocates to the nucleus, and releases its DNA payload. The delivered DNA forms episomes, leading to stable expression of the therapeutic gene.
03
Biological functions
Gene deliveryTransgene expressionCellular transduction
04
Disease associations
Gene therapy for inherited diseasesGene therapy for acquired diseases
05
Safety considerations
Pre-existing immunity to AAV serotypesImmunogenicity of AAV capsidOff-target gene expressionInsertional mutagenesis (rare)Limited packaging capacity
06
Biomarkers
AAV neutralizing antibody titers (pre-existing immunity)Transgene expression levels in target tissueImmune response to AAV capsid
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