Longitudinal EEG model detects antisense oligonucleotide treatment effect and increased UBE3A in Angelman syndrome

Angelman syndrome is a neurodevelopmental disorder caused by deficiency of the maternally inherited UBE3A gene in neurons. Antisense oligonucleotide therapies are under development to reinstate UBE3A protein production. Non-invasive biomarkers to detect target engagement and treatment response are n...

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Detalles Bibliográficos
Autores principales: Spencer, Elizabeth R., Shi, Wen, Komorowski, Robert W., Gilbert, James P., Ostrowski, Lauren M., Bird, Lynne M., Thibert, Ronald, Bao, Channa, Molloy, Fiona, Calhoun, Michael, Koirala, Samir, Jafar-nejad, Paymaan, Rigo, Frank, Kramer, Mark A., Chu, Catherine J.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Oxford University Press 2022
Materias:
Original Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9123847/
https://www.ncbi.nlm.nih.gov/pubmed/35611307
http://dx.doi.org/10.1093/braincomms/fcac106

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