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Human Treg responses allow sustained recombinant adeno-associated virus–mediated transgene expression

14 Nov 2013

Abstract excerpt

Recombinant adeno-associated virus (rAAV) vectors have shown promise for the treatment of several diseases; however, immune-mediated elimination of transduced cells has been suggested to limit and account for a loss of efficacy. To determine whether rAAV vector expression can persist long term, we administered rAAV vectors expressing normal, M-type α-1 antitrypsin (M-AAT) to AAT-deficient subjects at various...

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PMID: 24231351
DOI: 10.1172/jci70314

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