Adeno-Associated Virus Vectors

AAV vectors are used in several gene therapy applications because they can support gene delivery to selected tissues and may enable sustained transgene expression in appropriate settings.

The AAV capsid is an important target of immune recognition. Antibodies may bind capsid structures, and cellular immune responses may recognize capsid-derived peptides presented by transduced cells.

The magnitude and clinical significance of these responses vary with vector serotype, dose, administration route, tissue distribution, and individual immune characteristics.

AAV-based therapies therefore provide an important model for investigating the relationship between vector design, pre-existing immunity, and treatment-induced immune responses.