ISTH 2025 21 - 25 June 2025

Genomic integration of FVIII transgene in hepatocytes restores durable FVIII activity in vivo

Authors :

Jacob Lund¹; John Moore²; Carsten Dan Ley¹; Karl Agger²; Christopher William Smith¹; Jesper Tranekær Jørgensen¹; Michelle Scalley Kim²; Jazz Hallinan²; Kendall Sanson²; Rikke Hvid Lindecrona¹; Lene Diness Jensen¹; Nadin Jahnke²; Gert Bolt³; Erin Louise Brown²; Jonas Ahnfelt-Rønne¹; Esther Bloem³; Jonas Højfeldt²; Karina Thorn² and Gro Klitgaard Povlsen¹

Affiliations

View Details Hide Details

Keywords

Rare Blood Disorders

Congress oral presentation

Pipeline

F8-GE

Search Science Hub

Are you a healthcare professional?

Genomic integration of FVIII transgene in hepatocytes restores durable FVIII activity in vivo