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Enhanced liver gene transfer and evasion of preexisting humoral immunity with exosome-enveloped AAV vectors

Results from clinical trials of liver gene transfer for hemophilia demonstrate the potential of the adeno-associated virus (AAV) vector platform. However, to achieve therapeutic transgene expression, in some cases high vector doses are required, which are associated with a higher risk of triggering...

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Detalhes bibliográficos
Publicado no:Blood Adv
Main Authors: Meliani, Amine, Boisgerault, Florence, Fitzpatrick, Zachary, Marmier, Solenne, Leborgne, Christian, Collaud, Fanny, Simon Sola, Marcelo, Charles, Severine, Ronzitti, Giuseppe, Vignaud, Alban, van Wittenberghe, Laetitia, Marolleau, Beatrice, Jouen, Fabienne, Tan, Sisareuth, Boyer, Olivier, Christophe, Olivier, Brisson, Alain R., Maguire, Casey A., Mingozzi, Federico
Formato: Artigo
Idioma:Inglês
Publicado em: American Society of Hematology 2017
Assuntos:
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC5728288/
https://ncbi.nlm.nih.gov/pubmed/29296848
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1182/bloodadvances.2017010181
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