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Article

iPSC-hepatocyte organoids as a novel platform to predict AAV gene therapy efficacy

14 Apr 2025

Abstract excerpt

Adeno-associated virus (AAV) vectors are widely used in gene therapy, particularly for liver-targeted treatments. However, predicting human-specific outcomes, such as transduction efficiency and hepatotoxicity, remains challenging. Reliable in vitro models are urgently needed to bridge the gap between preclinical studies and clinical applications. This study presents the first comparative evaluation of AAV...

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PMID: 40927767
DOI: 10.1016/j.omtm.2025.101467

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