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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Foilsithe in:Hepatology
Main Authors: 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
Formáid: Artigo
Teanga:Inglês
Foilsithe: John Wiley and Sons Inc. 2017
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Original Articles
Rochtain Ar Líne:https://ncbi.nlm.nih.gov/pmc/articles/PMC5518300/
https://ncbi.nlm.nih.gov/pubmed/28318036
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1002/hep.29176
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