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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Bibliografiska uppgifter
I publikationen:Blood Adv
Huvudupphovsmän: 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
Materialtyp: Artigo
Språk:Inglês
Publicerad: American Society of Hematology 2017
Ämnen:
Gene Therapy
Länkar: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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