Ontology highlight
ABSTRACT:
SUBMITTER: Herzog RW
PROVIDER: S-EPMC5708148 | biostudies-literature | 2019 Aug
REPOSITORIES: biostudies-literature
Cellular immunology 20170529
After two decades of research, in vivo gene transfer with adeno-associated viral (AAV) vectors has now resulted in successful treatments and even cures for several human diseases. However, the potential for immune responses against the therapeutic gene products remains one of the concerns as this approach is broadened to more patients, diverse diseases, and target organs. Immune responses following gene transfer of coagulation factor IX (FIX) for the treatment of the bleeding disorder hemophilia ...[more]