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Development and Optimization of AAV hFIX Particles by Transient Transfection in an iCELLis <sup>®</sup> Fixed-Bed Bioreactor

27 May 2016

Abstract excerpt

Adeno-associated virus (AAV) vectors are increasingly popular in gene therapy because they are unassociated with human disease, replication dependent, and less immunogenic than other viral vectors and can infect a variety of cell types. These vectors have been used in over 130 clinical trials, and one AAV product has been approved for treatment of lipoprotein lipase deficiency in Europe. To meet the demand for...

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PMID: 27229773
DOI: 10.1089/hgtb.2016.021

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