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

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 betwe...

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Principais autores: Estelle Berreur, Giacomo Lazzaroni, Cyrill Roth, Marco Zihlmann, Martina Stirn, Ramona Matheis, Rebecca Xicluna, Ekaterina Breous-Nystrom, Adrian B. Roth
Formato: Artigo
Idioma:Inglês
Publicado em: Elsevier 2025-06-01
coleção:Molecular Therapy: Methods & Clinical Development
Assuntos:
AAV vectors
liver
iPSC
organoids
AAV transduction
AAV serotype
Acesso em linha:http://www.sciencedirect.com/science/article/pii/S2329050125000622
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