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...

Πλήρης περιγραφή

Αποθηκεύτηκε σε:
Λεπτομέρειες βιβλιογραφικής εγγραφής
Τόπος έκδοσης:Blood Adv
Κύριοι συγγραφείς: 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
Μορφή: Artigo
Γλώσσα:Inglês
Έκδοση: American Society of Hematology 2017
Θέματα:
Gene Therapy
Διαθέσιμο Online: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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