Displaying high-affinity ligands on adeno-associated viral vectors enables tumor cell-specific and safe gene transfer.

ABSTRACT: Gene transfer vectors derived from the adeno-associated virus (AAV) have recently received increasing attention due to substantial therapeutic benefit in several clinical trials. Nevertheless, their great potential for in vivo gene therapy can only be partially exploited owing to their broad tropism. Current cell surface targeting strategies expanded vector tropism towards transduction of cell types that are inefficiently infected naturally, but failed to restrict or fully re-direct AAV's tropism. Hypothesizing that this limitation can be overcome by equipping natural receptor-blinded AAV vectors with high-affinity ligands, we displayed designed ankyrin repeat proteins (DARPin) as VP2 fusion proteins on AAV capsids ablated for natural primary receptor binding. These second generation targe

SUBMITTER: Munch RC 

PROVIDER: S-EPMC3538307 | biostudies-literature | 2013 Jan

REPOSITORIES: biostudies-literature