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

16 Oct 2017

Abstract excerpt

vector genomes per kilogram of exo-AAV8 vectors, a staggering ∼1 log increase in hF.IX transgene expression was observed, leading to superior correction of clotting time. Enhanced liver expression was also associated with an increase in the frequency of regulatory T cells in lymph nodes. The efficiency of exo- and standard AAV8 vectors in evading preexisting NAbs to the capsid was then evaluated in a passive...

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PMID: 29296848
DOI: 10.1182/bloodadvances.2017010181

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