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Pharmacological read-through of R294X Mecp2 in a novel mouse model of Rett syndrome
Rett syndrome (RTT) is a neurodevelopmental disorder primarily caused by mutations in Methyl-CpG-binding Protein 2 (MECP2). More than 35% of affected individuals have nonsense mutations in MECP2. For these individuals, nonsense suppression has been suggested as a possible therapeutic approach. To as...
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| Publicado no: | Hum Mol Genet |
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| Main Authors: | , , , , |
| Formato: | Artigo |
| Idioma: | Inglês |
| Publicado em: |
Oxford University Press
2020
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| Assuntos: | |
| Acesso em linha: | https://ncbi.nlm.nih.gov/pmc/articles/PMC7471501/ https://ncbi.nlm.nih.gov/pubmed/32469049 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1093/hmg/ddaa102 |
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