Children's Hospital Research Foundation, Children's Hospital; Division of Molecular Medicine, Department of Pediatrics, and Department of Molecular Virology, Immunology, and Medical Genetics, College of Medicine and Public Health, The Ohio State University, Columbus, Ohio, USA

Correspondence: K. Reed Clark, Ph.D., Room W510, Children's Hospital, 700 Children's Drive, Columbus, Ohio 43205. E-mail: clarkr@pediatrics.ohio-state.edu

Abstract

Recent advances in recombinant adeno-associated virus vector production. Adeno-associated virus (AAV) is a replication-defective parvovirus that is being developed as a vector for human gene transfer. Recombinant AAV (rAAV) vectors are being proposed as a gene transfer vehicle for an array of human diseases. The recent interest in rAAV has been driven by the unexpected finding that these simple vectors can efficiently transduce a variety of postmitotic cells, resulting in long-lived, robust gene expression. However, a major obstacle to commonplace usage of rAAV vectors was the production in sufficient quantities for preclinical and human trials. Fortunately, several recent technological advances in vector production, purification, and titration have resulted in significant increases (>10-fold) in production capacity. Thus, there are several methods for the production of rAAV in excess of 10⁴ particles/cell, levels that should permit widespread use of this technology for clinical applications.