Adeno-associated Viral Vectors in Gene Therapy

Abstract

Recombinant vectors based on a nonpathogenic human parvovirus, the Adeno-associated virus 2 (AAV), have gained attention as a potentially safe and useful alternative to the more commonly used retroviral and adenoviral vectors. AAV vectors are currently in use in phase I/II clinical trials for gene therapy of a number of diseases such as cystic fibrosis, α-1 antitrypsin deficiency, muscular dystrophy, Batten disease and Parkinson disease. Several salient features of AAV vectors and the availability of promising results with animal models of human diseases provide the impetus to suggest that they will be used in potential gene therapy of a number of human diseases, both genetic and acquired, in not too distant a future.