Related Experiment Video
Updated: Jul 5, 2026
07:21
Production of Adeno-Associated Virus Vectors in Cell Stacks for Preclinical Studies in Large Animal Models
Published on: June 30, 2021
Manufacturing and characterizing AAV-based vectors for use in clinical studies
1Center for Cellular and Molecular Therapeutics, Children's Hospital of Philadelphia, Philadelphia, PA, USA. wrightf@email.chop.edu
Gene Therapy
|April 18, 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.
