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A scalable method for the production of high-titer and high-quality adeno-associated type 9 vectors using the HSV platform

1 Jan 2016

Abstract excerpt

Recombinant adeno-associated vectors based on serotype 9 (rAAV9) have demonstrated highly effective gene transfer in multiple animal models of muscular dystrophies and other neurological indications. Current limitations in vector production and purification have hampered widespread implementation of clinical candidate vectors, particularly when systemic administration is considered. In this study, we describe a...

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PMID: 27222839
DOI: 10.1038/mtm.2016.31

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