Cargando…
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...
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: | |
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 |
Ejemplares similares
-
Longitudinal EEG model detects antisense oligonucleotide treatment effect and increased UBE3A in Angelman syndrome
por: Spencer, Elizabeth R., et al.
Publicado: (2022) -
Antisense oligonucleotide therapy for KCNT1 encephalopathy
por: Burbano, Lisseth Estefania, et al.
Publicado: (2022) -
Sarm1 haploinsufficiency or low expression levels after antisense oligonucleotides delay programmed axon degeneration
por: Gould, Stacey Anne, et al.
Publicado: (2021) -
The atlas of RNase H antisense oligonucleotide distribution and activity in the CNS of rodents and non-human primates following central administration
por: Jafar-nejad, Paymaan, et al.
Publicado: (2020) -
Antisense oligonucleotide treatment rescues UBE3A expression and multiple phenotypes of an Angelman syndrome mouse model
por: Milazzo, Claudia, et al.
Publicado: (2021)