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Assessing the requirements of prenatal UBE3A expression for rescue of behavioral phenotypes in a mouse model for Angelman syndrome

Abstract Background Angelman syndrome (AS) is a rare neurodevelopmental disorder caused by the loss of functional ubiquitin protein ligase E3A (UBE3A). In neurons, UBE3A expression is tightly regulated by a mechanism of imprinting which suppresses the expression of the paternal UBE3A allele. Promisi...

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Principais autores: Monica Sonzogni, Peipei Zhai, Edwin J. Mientjes, Geeske M. van Woerden, Ype Elgersma
Formato: Artigo
Idioma:Inglês
Publicado: BMC 2020-09-01
Series:Molecular Autism
Assuntos:
Angelman syndrome
UBE3A
Mouse model
Behavior
Critical period
ASO therapy
Acceso en liña:http://link.springer.com/article/10.1186/s13229-020-00376-9
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