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AntimiR targeting of microRNA-134 reduces seizures in a mouse model of Angelman syndrome

Angelman syndrome (AS) is a severe neurodevelopmental disorder featuring ataxia, cognitive impairment, and drug-resistant epilepsy. AS is caused by mutations or deletion of the maternal copy of the paternally imprinted UBE3A gene, with current precision therapy approaches focusing on re-expression o...

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Detalles Bibliográficos
Autores principales: Campbell, Aoife, Morris, Gareth, Sanfeliu, Albert, Augusto, Joana, Langa, Elena, Kesavan, Jaideep C., Nguyen, Ngoc T., Conroy, Ronan M., Worm, Jesper, Kielpinski, Lukasz, Jensen, Mads Aaboe, Miller, Meghan T., Kremer, Thomas, Reschke, Cristina R., Henshall, David C.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society of Gene & Cell Therapy 2022
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9092865/
https://www.ncbi.nlm.nih.gov/pubmed/35592499
http://dx.doi.org/10.1016/j.omtn.2022.04.009