Author

Geneviève Gourdon

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Open access Jul 2026

Cortical organoids from congenital DM1 PSCs reveal MBNL-dependent corticogenesis defects and enable preclinical testing of therapeutic compounds

Cortical organoids are established as a robust human model of CDM-associated neurodevelopmental defects, uncover MBNL-dependent mechanisms underlying early corticogenesis impairment and demonstrate the utility of this platform for translational therapeutic discovery in DM1.

Azania Abatan, Jérôme Polentes, M. Bouquier et al. · bioRxiv · 0 citations

Open access Jul 2026

Fatty-acid-based antimiR-23b delivery in the DMSXL model: A potential therapeutic strategy for brain dysfunction in myotonic dystrophy type 1

This study evaluates the therapeutic potential of the lipid-conjugated antimiR-23b, X82108, designed to promote MBNL1/2 upregulation through inhibition of miR-23b, and highlights X82108 as a promising systemic therapy for DM1.

D. Piqueras-Losilla, Andrea García-Rey, Aline Huguet-Lachon et al. · Cell Reports Medicine · 0 citations