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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...
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. |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
American Society of Gene & Cell Therapy
2022
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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 |
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