Author

C. Vorhees

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Open access Jul 2026

FMR1 gene therapy restores translationally relevant phenotypes in a mouse model for fragile X syndrome.

It is shown that FMR1 gene therapy using delivery routes and vehicles approved for clinical use improves core phenotypes in a mouse model for FXS, and suggests that a dual route may be needed to achieve full brain coverage.

Richard K Lacher, Kari Henson, Lindsay N Wathen et al. · Gene Therapy · 0 citations