Antisense oligonucleotide therapy rescues disturbed brain rhythms and sleep in juvenile and adult mouse models of Angelman syndrome

UBE3A encodes ubiquitin protein ligase E3A, and in neurons its expression from the paternal allele is repressed by the UBE3A antisense transcript (UBE3A-ATS). This leaves neurons susceptible to loss-of-function of maternal UBE3A. Indeed, Angelman syndrome, a severe neurodevelopmental disorder, is ca...

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Detalles Bibliográficos
Autores principales: Lee, Dongwon, Chen, Wu, Kaku, Heet Naresh, Zhuo, Xinming, Chao, Eugene S, Soriano, Armand, Kuncheria, Allen, Flores, Stephanie, Kim, Joo Hyun, Rivera, Armando, Rigo, Frank, Jafar-nejad, Paymaan, Beaudet, Arthur L, Caudill, Matthew S, Xue, Mingshan
Formato: Online Artículo Texto
Lenguaje:English
Publicado: eLife Sciences Publications, Ltd 2023
Materias:
Neuroscience
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9904759/
https://www.ncbi.nlm.nih.gov/pubmed/36594817
http://dx.doi.org/10.7554/eLife.81892

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9904759/
https://www.ncbi.nlm.nih.gov/pubmed/36594817
http://dx.doi.org/10.7554/eLife.81892

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