AAV Vector Immunogenicity in Humans: A Long Journey to Successful Gene Transfer.

ABSTRACT: Gene therapy with adeno-associated virus (AAV) vectors has demonstrated safety and long-term efficacy in a number of trials across target organs, including eye, liver, skeletal muscle, and the central nervous system. Since the initial evidence that AAV vectors can elicit capsid T cell responses in humans, which can affect the duration of transgene expression, much progress has been made in understanding and modulating AAV vector immunogenicity. It is now well established that exposure to wild-type AAV results in priming of the immune system against the virus, with development of both humoral and T cell immunity. Aside from the neutralizing effect of antibodies, the impact of pre-existing immunity to AAV on gene transfer is still poorly understood. Herein, we review data emerging from clini

SUBMITTER: Verdera HC 

PROVIDER: S-EPMC7054726 | biostudies-literature | 2020 Mar

REPOSITORIES: biostudies-literature