What is the prevalence and profile of neutralizing antibodies to AAV2 and AAV8 in human subjects aged 0 to 18 years?
The prevalence of neutralizing antibodies to AAV vectors fluctuates significantly from birth through adolescence, which may impact the timing and efficacy of AAV-mediated gene therapies in pediatric populations.
Neutralizing antibodies (NAb) to an adeno-associated virus (AAV) vector due to previous natural infection with wild-type AAV can significantly limit gene transfer. NAb titers to AAV serotype 2 (AAV2) and AAV8 in human subjects (0 to 18 years) were studied. NAb prevalence is moderate at birth, decreases markedly from 7 to 11 months, and then progressively increases through childhood and adolescence.
Calcedo et al. (Wed,) studied this question.