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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Detalhes bibliográficos
Publicado no: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
Formato: Artigo
Idioma:Inglês
Publicado em: John Wiley and Sons Inc. 2017
Assuntos:
Original Articles
Acesso em linha: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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