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Author

A. R. Batista

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Open access Jul 2026

AAV-mediated gene therapy demonstrates phenotypic rescue in a mouse model of Cockayne syndrome

Treatment with AAV9-CSA resulted in a significant increase in life span, and broad distribution of human CSA in the brain and heart, without evidence of vector-related toxicity, and paves the way for clinical translation of an AAV gene therapy for patients with CS into humans.

A. R. Batista, Aine C. Scholand, William S. Callahan et al. · 0 citations

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