Antisense oligonucleotide treatment rescues UBE3A expression and multiple phenotypes of an Angelman syndrome mouse model

Angelman syndrome (AS) is a severe neurodevelopmental disorder for which only symptomatic treatment with limited benefits is available. AS is caused by mutations affecting the maternally inherited ubiquitin protein ligase E3A (UBE3A) gene. Previous studies showed that the silenced paternal Ube3a gen...

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Detalles Bibliográficos
Autores principales: Milazzo, Claudia, Mientjes, Edwin J., Wallaard, Ilse, Rasmussen, Søren Vestergaard, Erichsen, Kamille Dumong, Kakunuri, Tejaswini, van der Sman, A.S. Elise, Kremer, Thomas, Miller, Meghan T., Hoener, Marius C., Elgersma, Ype
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society for Clinical Investigation 2021
Materias:
Research Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8410092/
https://www.ncbi.nlm.nih.gov/pubmed/34369389
http://dx.doi.org/10.1172/jci.insight.145991

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8410092/
https://www.ncbi.nlm.nih.gov/pubmed/34369389
http://dx.doi.org/10.1172/jci.insight.145991

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