Recombinant adeno-associated virus vectors for gene therapy

Abstract

Recombinant adeno-associated virus (rAAV) vectors are based on a non-pathogenic human parvovirus (AAV) that is unique in its ability to persist in human cells without causing any pathologic effects. Studies of the potential barriers to rAAV-mediated transduction of relatively resistant cells has led to an understanding of the mechanisms of cell attachment and entry, cytoplasmic translocation, nuclear entry, conversion to active double-stranded DNA, activation of transcription and establishment of persistent molecular forms. Each of these areas is individually discussed, as are recent applications in vivo in preclinical models and clinical trials.