Ontology highlight
ABSTRACT:
SUBMITTER: Samelson-Jones BJ
PROVIDER: S-EPMC7256432 | biostudies-literature | 2020 Jun
REPOSITORIES: biostudies-literature
Samelson-Jones Benjamin J BJ Finn Jonathan D JD Favaro Patricia P Wright J Fraser JF Arruda Valder R VR
Molecular therapy. Methods & clinical development 20200512
Adeno-associated virus (AAV) vector gene therapy is a promising treatment for a variety of genetic diseases, including hemophilia. Systemic administration of AAV vectors is associated with a cytotoxic immune response triggered against AAV capsid proteins, which if untreated can result in loss of transgene expression. Immunosuppression (IS) with corticosteroids has limited transgene loss in some AAV gene therapy clinical trials, but was insufficient to prevent loss in other studies. We used a non ...[more]