Preclinical assessment of an optimized AAV-FVIII vector in mice and non-human primates for the treatment of hemophilia A

Abstract excerpt

Extensive clinical data from liver-mediated gene therapy trials have shown that dose-dependent immune responses against the vector capsid may impair or even preclude transgene expression if not managed successfully with prompt immune suppression. The goal of this preclinical study was to generate an adeno-associated viral (AAV) vector capable of expressing therapeutic levels of B-domain deleted factor VIII...

Topics

Share this publication in a Topic to start or enrich a Post.

• Share to CAR-T cell therapy research
• Share to Hemophilia Treatment and Research
• Share to Virus-based gene therapy research