1. ¹Center for Cellular and Molecular Therapeutics, Children's Hospital of Philadelphia, Philadelphia, PA, USA
2. ²Department of Pathology and Laboratory Medicine, University of Pennsylvania School of Medicine, Philadelphia, PA, USA

Correspondence: Dr JF Wright, Department of Pathology and Laboratory Medicine, The Children's Hospital of Philadelphia, University of Pennsylvania School of Medicine, 3615 Civic Center Blvd, ARC1216C, Philadelphia, PA 19104, USA. E-mail: wrightf@email.chop.edu

Received 27 February 2008; Revised 5 March 2008; Accepted 6 March 2008; Published online 17 April 2008.

Abstract

Recombinant adeno-associated virus (AAV)-based vectors expressing therapeutic gene products have shown great promise for human gene therapy. A major challenge for translation of promising research to clinical development is the manufacture and certification of AAV vectors for clinical use. This review summarizes relevant aspects of current Good Manufacturing Practice, focusing on considerations and challenges specific for recombinant AAV.