Unlocking the potential of allele-specific siRNA therapy: A novel approach for Crouzon syndrome.

Mol Ther Nucleic Acids

Pediatric Neurosurgery Unit, Centre de Référence Craniosténose - Hôpital Femme Mère Enfant, Groupement Hospitalier Est - Hospices Civils de Lyon, INSERM UMR 1033, Faculté de Médecine Lyon-Est, Université Claude Bernard Lyon 1, Lyon, France.

Published: March 2025

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http://www.ncbi.nlm.nih.gov/pmc/articles/PMC11889969PMC
http://dx.doi.org/10.1016/j.omtn.2025.102484DOI Listing

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