Author

Christina Gross

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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

Open access Jul 2026

The m6A-mediated epi-transcriptomic dysregulation drives synaptic dysfunction in fragile X syndrome.

It is demonstrated that FMRP deficiency drives the aberrant translational upregulation of core m6A writers, a causal relationship definitively validated using CGG-corrected isogenic control lines and suggest a promising avenue for m6A-targeted therapies.

Lu Lu, Avijite Kumer Sarkar, Lan Dao et al. · Molecular Psychiatry · 0 citations