Factor IX assay discrepancies in the setting of liver gene therapy using a hyperfunctional variant factor IX-Padua.

Mary M RobinsonLindsey A GeorgeMarcus E CarrBenjamin J Samelson-JonesValder R ArrudaJohn E MurphyDenis RybinJeremy RuponKatherine A HighStefan Tiefenbacher

Published in: Journal of thrombosis and haemostasis : JTH (2021)

Assay-based differences in FIX:C were observed for fidanacogene elaparvovec transgene product and rHFIX-Padua protein, suggesting the variable FIX:C determined with different assay reagents is inherent to the FIX-Padua protein and is not specific to gene therapy-derived FIX-Padua.

Keyphrases

gene therapy
high throughput
protein protein
binding protein
small molecule