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Gene therapy: challenges in cell culture scale-up

7 Apr 2022

Abstract excerpt

Gene therapy is designed to cure various diseases resulting from genetic defects. Currently, recombinant adeno-associated viral vectors (rAAV) are the vehicles of choice for therapeutic gene delivery in vivo. To date, manufacturing sufficient rAAV product to meet rapidly expanding clinical demand remains a bottleneck in the industry. In the past decade, multiple production platforms have been rapidly implemented...

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PMID: 35398708
DOI: 10.1016/j.copbio.2022.102721

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