rAAV Fbxo7 gene therapy rescues the progressive nigrostriatal pathology in a mouse model of juvenile parkinsonism.

Acta Neuropathol Commun · 2026 · PMC13261993 · PMID 41992302

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highly significantno p-value reported

However, despite the much higher viral load, the EV treatment did not approach the highly significant, complete phenotypic rescue (restoring TH levels to > 110%) observed with the much lower-dose Fbxo7 vector.

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