Establishment of a novel cell line for the enhanced production of recombinant adeno-associated virus vectors for gene therapy.

Abstract

Adeno-associated viral (AAV) vectors show great promise because of their excellent safety profile; however, pre-existing immune responses have necessitated the administration of high titer AAV, posing a significant challenge to the advancement of gene therapy involving AAV vectors. Recombinant AAV vectors contain minimum viral proteins necessary for their… (More)
DOI: 10.1089/hum.2014.041

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