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Towards a therapy for Angelman syndrome by reduction of a long non-coding RNA

Angelman syndrome (AS) is a single gene disorder characterized by intellectual disability, developmental delay, behavioral uniqueness, speech impairment, seizures, and ataxia(1,2). It is caused by maternal deficiency of the imprinted gene UBE3A, encoding an E3 ubiquitin ligase(3-5). All patients car...

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Detalles Bibliográficos
Autores principales: Meng, Linyan, Ward, Amanda J., Chun, Seung, Bennett, C. Frank, Beaudet, Arthur L., Rigo, Frank
Formato: Online Artículo Texto
Lenguaje:English
Publicado: 2014
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4351819/
https://www.ncbi.nlm.nih.gov/pubmed/25470045
http://dx.doi.org/10.1038/nature13975