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 assembly and gene delivery functions. During the process of AAV assembly and production, AAV vectors acquire, inherently and submissively, various cellular proteins, but the identity of these proteins is poorly characterized. We reason that by identifying host cell proteins inherently associated with AAV vectors we may better understand the contribution of cellular components to AAV vector assembly and, ultimately, may improve the product

SUBMITTER: Satkunanathan S 

PROVIDER: S-EPMC4236031 | biostudies-literature | 2014 Nov

REPOSITORIES: biostudies-literature