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.
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We thank Drs Guang Qu, Olga Zelenaia and Bernd Hauck for helpful discussions and review of the manuscript.
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Wright, J. Manufacturing and characterizing AAV-based vectors for use in clinical studies. Gene Ther 15, 840–848 (2008). https://doi.org/10.1038/gt.2008.65
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DOI: https://doi.org/10.1038/gt.2008.65
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