AAV vectors are used in gene therapy products for treatment of genetic diseases. The high natural prevalence of AAV wild-type viruses has resulted in a high frequency of capsid-directed humoral immunity in human populations and many animal models, which may interfere with the therapeutic efficiency of a therapy if not identified and managed properly.
This case study describes the development of an anti-AAV immune assay for use in preclinical studies using cynomolgus monkeys, with the ability to screen for pre-existing AAV immunity and to identify low-responding individuals to maximize the likelihood of successful transduction.