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Autophagy determines efficiency of liver‐directed gene therapy with adeno‐associated viral vectors

Use of adeno‐associated viral (AAV) vectors for liver‐directed gene therapy has shown considerable success, particularly in patients with severe hemophilia B. However, the high vector doses required to reach therapeutic levels of transgene expression caused liver inflammation in some patients that s...

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Detalles Bibliográficos
Autores principales:	Hösel, Marianna, Huber, Anke, Bohlen, Susanne, Lucifora, Julie, Ronzitti, Giuseppe, Puzzo, Francesco, Boisgerault, Florence, Hacker, Ulrich T., Kwanten, Wilhelmus J., Klöting, Nora, Blüher, Matthias, Gluschko, Alexander, Schramm, Michael, Utermöhlen, Olaf, Bloch, Wilhelm, Mingozzi, Federico, Krut, Oleg, Büning, Hildegard
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	John Wiley and Sons Inc. 2017
Materias:	Original Articles
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5518300/ https://www.ncbi.nlm.nih.gov/pubmed/28318036 http://dx.doi.org/10.1002/hep.29176

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