Modulation of the liver immune microenvironment by the adeno-associated virus serotype 8 gene therapy vector.

ABSTRACT: Adeno-associated viruses (AAVs) are emerging as one of the vehicles of choice for gene therapy. However, the potential immunogenicity of these vectors is a major limitation of their use, leading to the necessity of a better understanding of how viral vectors engage the innate immune system. In this study, we demonstrate the immune response mediated by an AAV vector in a mouse model. Mice were infected intravenously with 4 × 10¹² copies (cp)/kg of AAV8, and the ensuing immune response was analyzed using intravital microscopy during a period of weeks. Administration of AAV8 resulted in the infection of hepatocytes, and this infection led to a moderate, but significant, activation of the immune system in the liver. This host immune response involved platelet aggregation, neutrophil

SUBMITTER: Carestia A 

PROVIDER: S-EPMC7750493 | biostudies-literature | 2021 Mar

REPOSITORIES: biostudies-literature