Ontology highlight
ABSTRACT:
SUBMITTER: Weber A
PROVIDER: S-EPMC6039963 | biostudies-literature | 2018 Sep
REPOSITORIES: biostudies-literature
Weber Alfred A Engelmaier Andrea A Voelkel Dirk D Pachlinger Robert R Scheiflinger Friedrich F Monahan Paul E PE Rottensteiner Hanspeter H
Molecular therapy. Methods & clinical development 20180628
The description of hyper-functional factor IX (FIX) Padua triggered the development of BAX 335, an AAV8-based hemophilia B gene therapy vector designed to compensate for low FIX protein expression levels by expressing the FIX Padua variant, thereby reducing the exposure to viral vector. The presence of inactive FIX protein at baseline hindered conventional FIX:Ag ELISA from contributing to a more profound understanding of clinical data from the BAX 335 Phase 1/2 study (ClinicalTrials.gov: NCT016 ...[more]