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Organoids and microphysiological systems: Promising models for accelerating AAV gene therapy studies

26 Sept 2022

Abstract excerpt

The FDA has predicted that at least 10-20 gene therapy products will be approved by 2025. The surge in the development of such therapies can be attributed to the advent of safe and effective gene delivery vectors such as adeno-associated virus (AAV). The enormous potential of AAV has been demonstrated by its use in over 100 clinical trials and the FDA's approval of two AAV-based gene therapy products. Despite its...

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PMID: 36225917
DOI: 10.3389/fimmu.2022.1011143

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