AAV vectors trigger DNA damage response-dependent pro-inflammatory signalling in human iPSC-derived CNS models and mouse brain

AAV vectors trigger DNA damage response-dependent pro-inflammatory signalling in human iPSC-derived CNS models and mouse brain

Abstract Adeno-associated viral (AAV) vector-based gene therapy is gaining foothold as treatment for genetic neurological diseases with encouraging clinical results. Nonetheless, dose-dependent adverse events have emerged in recent clinical trials through mechanisms that remain unclear. We have mode...

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Main Authors: Helena Costa-Verdera, Vasco Meneghini, Zachary Fitzpatrick, Monah Abou Alezz, Emily Fabyanic, Xin Huang, Yulia Dzhashiashvili, Avantika Ahiya, Elisabeth Mangiameli, Erika Valeri, Giovanni Crivicich, Silvia Piccolo, Ivan Cuccovillo, Roberta Caccia, Ying Kai Chan, Bérangère Bertin, Giuseppe Ronzitti, Esteban A. Engel, Ivan Merelli, Federico Mingozzi, Angela Gritti, Klaudia Kuranda, Anna Kajaste-Rudnitski
Format: Article
Language:English
Published: Nature Portfolio 2025-04-01
Series:Nature Communications
Online Access:https://doi.org/10.1038/s41467-025-58778-3
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https://doi.org/10.1038/s41467-025-58778-3

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