Prospects for the development of targeted adeno-associated virus (AAV) vector systems

Gene transfer vectors based on adeno-associated virus (AAV) show great promise for human gene therapy. However, the efficiency of AAV-mediated gene delivery to different cell types can vary greatly, The ability to mediate targeted and specific delivery of therapeutics remains one of the most important hurdles in effectively treating human disease. Targeted AAV vectors are being developed with the goal of overcoming this hurdle, Currently, AAV vector targeting has been achieved via transcriptional regulation and with bispecific antibodies or specific ligands that are capable of mediating novel interactions between the AAV vector and specific cell surface receptors, This review presents an overview of current and potential methods for developing targeted AAV vectors. Future studies will determine if the efficacy of therapeutic AAV vectors is enhanced using these modifications.