Work

595. Enhanced Liver Transduction and Efficient Protection from Pre-Existing Neutralizing Antibodies with Exosome-Associated AAV8 Vectors

Year: 2016

Type: article

Abstract: Adeno-associated virus (AAV) based vectors are ideal tools for in vivo gene transfer. The excellent safety profile and the highly efficient targeting of hepatocytes in vivo have been demonstrated in both preclinical studies and clinical trials. Although AAV vectors are not strongly immunogenic, they can give rise to both cellular and humoral immune responses. Additionally, as a result of exposure to wild-type AAV, an important fraction of humans harbor pre-existing neutralizing antibodies against the viral capsid, which can prevent successful transduction by AAV vectors.

Source: Molecular Therapy

DOI: 10.1016/s1525-0016(16)33403-7