Evaluation of Novel Enhancer Compounds in Gentamicin-Mediated Readthrough of Nonsense Mutations in Rett Syndrome

Rett syndrome (RTT), a severe X-linked neurodevelopmental disorder, is primarily caused by mutations in the methyl CpG binding protein 2 gene (MECP2). Over 35% RTT patients carry nonsense mutation in MECP2, making it a suitable candidate disease for nonsense suppression therapy. In our previous stud...

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Detalles Bibliográficos
Autores principales: Wong, Keit Men, Wegener, Eike, Baradaran-Heravi, Alireza, Huppke, Brenda, Gärtner, Jutta, Huppke, Peter
Formato: Online Artículo Texto
Lenguaje:English
Publicado: MDPI 2023
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10380790/
https://www.ncbi.nlm.nih.gov/pubmed/37511424
http://dx.doi.org/10.3390/ijms241411665

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10380790/
https://www.ncbi.nlm.nih.gov/pubmed/37511424
http://dx.doi.org/10.3390/ijms241411665

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