ISTH 2025
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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
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Keywords
Rare Blood Disorders
Congress oral presentation
Pipeline