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

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 (<i>MECP2</i>). Over 35% RTT patients carry nonsense mutation in <i>MECP2</i>, making it a suitable candidate disease for nonsense sup...

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Bibliographic Details
Main Authors: Keit Men Wong, Eike Wegener, Alireza Baradaran-Heravi, Brenda Huppke, Jutta Gärtner, Peter Huppke
Format: Article
Language:English
Published: MDPI AG 2023-07-01
Series:International Journal of Molecular Sciences
Subjects:
Rett syndrome
readthrough
aminoglycosides
nonsense suppression therapy
methyl-CpG-binding protein
Online Access:https://www.mdpi.com/1422-0067/24/14/11665

Internet

https://www.mdpi.com/1422-0067/24/14/11665

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