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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| Autors principals: | , , , , , , , , |
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| Format: | Artigo |
| Idioma: | Inglês |
| Publicat: |
Elsevier
2025-06-01
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| Col·lecció: | Molecular Therapy: Methods & Clinical Development |
| Matèries: | |
| Accés en línia: | http://www.sciencedirect.com/science/article/pii/S2329050125000622 |
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