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

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 techno...

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Detalles Bibliográficos
Autores principales:	Baruteau, Julien, Waddington, Simon N., Alexander, Ian E., Gissen, Paul
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	Springer Netherlands 2017
Materias:	Ssiem 2016
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5500673/ https://www.ncbi.nlm.nih.gov/pubmed/28567541 http://dx.doi.org/10.1007/s10545-017-0053-3

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