Evaluation of Novel Enhancer Compounds in Gentamicin-Mediated Readthrough of Nonsense Mutations in Rett Syndrome
Authors Wong KM, Wegener E, Baradaran-Heravi A, Huppke B, Gärtner J, Huppke P Journal International Journal of Molecular Sciences Citation Int J Mol Sci. 2023 Jul 19;24(14):11665. Abstract Rett syndrome (RTT), a severe X-linked neurodevelopmental disorder, is primarily caused by mutations in the methyl CpG binding protein 2 gene (MECP2).



