Unraveling the Complex Story of Immune Responses to AAV Vectors Trial After Trial.

ABSTRACT: Over the past decade, vectors derived from adeno-associated virus (AAV) have established themselves as a powerful tool for in vivo gene transfer, allowing long-lasting and safe transgene expression in a variety of human tissues. Nevertheless, clinical trials demonstrated how B and T cell immune responses directed against the AAV capsid, likely arising after natural infection with wild-type AAV, might potentially impact gene transfer safety and efficacy in patients. Seroprevalence studies have evidenced that most individuals carry anti-AAV neutralizing antibodies that can inhibit recombinant AAV transduction of target cells following in vivo administration of vector particles. Likewise, liver- and muscle-directed clinical trials have shown that capsid-reactive memory CD8⁺ T cells

SUBMITTER: Vandamme C 

PROVIDER: S-EPMC5649404 | biostudies-literature | 2017 Nov

REPOSITORIES: biostudies-literature