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Gene therapy for monogenic liver diseases: clinical successes, current challenges and future prospects

31 May 2017

Abstract excerpt

Over the last decade, pioneering liver-directed gene therapy trials for haemophilia B have achieved sustained clinical improvement after a single systemic injection of adeno-associated virus (AAV) derived vectors encoding the human factor IX cDNA. These trials demonstrate the potential of AAV technology to provide long-lasting clinical benefit in the treatment of monogenic liver disorders. Indeed, with more than...

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PMID: 28567541
DOI: 10.1007/s10545-017-0053-3

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