Effective Reduction of Transgene-Specific Immune Response With rAAV Vectors Co-Expressing miRNA-UL112-5p or ERAP1 shRNA.

ABSTRACT: Recombinant adeno-associated virus (rAAV) has emerged as one of the best gene delivery vectors for human gene therapy in vivo. However, the clinical efficacy of rAAV gene therapy is often hindered by the host immune response against its transgene products. Endoplasmic reticulum aminopeptidase 1 (ERAP1) is specialised to process peptides presented by class I molecules of major histocompatibility complex. Therefore, we hypothesise that modulation of the ERAP1 activity in rAAV transduced cells may be favoured to evade immune response against transgene products. In this study, we incorporated either miRNA-UL112-5p or ERAP1 shRNA into rAAV vectors expressing full-length ovalbumin (OVA) as a model antigen, and evaluated their effects for antigen presentation, cellular and humour immune response

SUBMITTER: Huang X 

PROVIDER: S-EPMC11740984 | biostudies-literature | 2025 Jan

REPOSITORIES: biostudies-literature