Safety of adeno-associated virus gene therapy vectors: a current evaluation

Abstract

An increasing number of strategies for molecular treatment of disease rely on the adeno-associated virus (AAV) as a therapeutic gene delivery vector. One of the most attractive features of this viral DNA vector is the perceived safety of AAV gene delivery. Recent applications in human clinical trials support the safety record established in preclinical trials, with evidence of gene transfer in the absence of cellular immune responses or tissue disturbance. Nevertheless, many aspects of the biology of the wild type AAV and its derivatives are still being explored. While the therapeutic potential of novel recombinant AAV therapeutics appears promising, recent insights suggest aspects of their pharmacokinetics, biodistribution and toxicity that require consideration to achieve the safest application of these molecular medicines.