BIC 2025 12 - 14 September 2025

Endonuclease-mediated genomic insertion of AAV-delivered FVIII transgene into hepatocyte safe-harbor locus restores durable FVIII activity in vivo

Authors :

Jacob Lund¹; Carsten Dan Ley¹; Karl Agger²; Christopher William Smith¹; Jesper Tranekjær Jørgensen¹; Michelle Scalley Kim²; Jazz Hallinan²; Kendall Sanson²; Rikke Hvid Lindecrona³; Lene Diness Jensen³; Nadin Jahnke²; Gert Bolt²; Erin Louise Brown²; Jonas Ahnfelt-Rønne³; Esther Bloem²; Jonas Højfeldt²; Karina Thorn² and Gro Klitgaard Povlsen¹

Affiliations

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Keywords

Haemophilia

F8-GE

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Endonuclease-mediated genomic insertion of AAV-delivered FVIII transgene into hepatocyte safe-harbor locus restores durable FVIII activity in vivo