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Article

Cas9 gene therapy for Angelman syndrome traps Ube3a-ATS long non-coding RNA.

Nature - 1 Nov 2020

Wolter Justin M, Mao Hanqian, Fragola Giulia, Simon Jeremy M, Krantz James L, Bazick Hannah O, Oztemiz Baris, Stein Jason L, Zylka Mark J

Abstract excerpt

Angelman syndrome (AS) is a severe neurodevelopmental disorder caused by a mutation or deletion of the maternally inherited UBE3A allele. In neurons, the paternally inherited UBE3A allele is silenced in cis by a long non-coding RNA called UBE3A-ATS. Here, as part of a systematic screen, we found that Cas9 can be used to activate ('unsilence') paternal Ube3a in cultured mouse and human neurons when targeted to...

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Identifiers

PMID: 33087932
DOI: 10.1038/s41586-020-2835-2
PMCID: PMC8020672

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