Innate and adaptive AAV-mediated immune responses in a mouse model of Duchenne muscular dystrophy

High systemic doses of adeno-associated viruses (AAVs) have been associated with immune-related serious adverse events (SAEs). Although AAV was well tolerated in preclinical models, SAEs were observed in clinical trials, indicating the need for improved preclinical models to understand AAV-induced i...

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Main Authors: Michael R. Emami, Alejandro Espinoza, Courtney S. Young, Feiyang Ma, Philip K. Farahat, Philip L. Felgner, Jeffrey S. Chamberlain, Xiangmin Xu, April D. Pyle, Matteo Pellegrini, S. Armando Villalta, Melissa J. Spencer
Format: Article
Language:English
Published: Elsevier 2023-09-01
Series:Molecular Therapy: Methods & Clinical Development
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Online Access:http://www.sciencedirect.com/science/article/pii/S232905012300092X