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AAV-Based Gene Therapy: Opportunities, Risks, and Scale-Up Strategies

26 Aug 2025

Abstract excerpt

Currently, the development of adeno-associated virus (AAV)-based gene therapy is a promising method for treating various diseases and is gaining increasing popularity. However, the use of AAV has certain drawbacks and faces limitations such as immune responses and an increased risk of insertional mutagenesis, which have not always been adequately considered in the context of AAV therapy. Moreover, a significant...

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PMID: 40943204
DOI: 10.3390/ijms26178282

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