A single administration of AAV5-hFIX in newborn, juvenile and adult mice leads to stable hFIX expression up to 18 months after dosing

Jaap Twisk, uniQure Biopharma B.V.; Liesbeth Heijink, uniQure Biopharma B.V.; Lisa Spronck, uniQure Biopharma B.V.; Martin de Haan, uniQure Biopharma B.V.; Richard van Logtenstein, uniQure Biopharma B.V.; Sander van Deventer, uniQure Biopharma B.V.; Valerie Ferreira, uniQure Biopharma B.V.