117. HSPG Binding Properties of Adeno-Associated Virus Retargeting Mutants and Consequences for Their In Vivo Tropism

Different technologies have been developed to control or redirect tropism of adeno-associated virus of type 2 (AAV-2) by genetic capsid modification. Many of these approaches are based on the introduction of peptide sequences on the viral capsid in order to provide the vector with the ability to bind particular cellular receptors. To obtain selective vectors however, simultaneous abrogation of capsid usage of natural receptors is essential. Insertions at amino acid position 587 of the major capsid protein VP1 ablated the heparan sulphate proteoglycan binding of wild-type AAV in some, but not all previously described AAV targeting vectors.