Article
Epigenetic editing alleviates Angelman syndrome phenotype in mice by unsilencing paternal Ube3a
17 Sept 2024
Abstract excerpt
Angelman syndrome (AS) is characterized by severe neurodevelopmental disorders caused by abnormalities in the maternally inherited UBE3A gene, leading to a complete loss of UBE3A specifically in neurons, where the intact paternal UBE3A is silenced by an endogenous antisense long-noncoding RNA known as UBE3A-ATS . The expression of UBE3A-ATS is regulated by the imprinting center Snrpn ( Snrpn -IC) 1 , which is...
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