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Vector Development

High expression levels of alpha 1-antitrypsin are mediated by adeno-associated virus

Published in Vaccine Weekly, March 8th, 2006

High levels of persistent expression of alpha 1-antitrypsin are mediated by the nonhuman primate serotype rh.10 adeno-associated virus despite preexisting immunity to common human adeno-associated viruses.

According to researchers in the United States, "Alpha 1-antitrypsin (alpha 1AT) deficiency is a genetic disorder causing emphysema if serum alpha 1AT levels are <570 mcg/mL. We have shown that intrapleural administration of an AAV5 alpha 1AT vector yielded persistent therapeutic alpha 1AT serum levels."

"Since anti-AAV2 and -AAV5 antibodies prevalent in humans may limit the use of these common serotypes in gene therapy, we screened 25 AAV...

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