Towards a therapy for Angelman syndrome by targeting a long non-coding RNA

Linyan Meng, Amanda J. Ward, Seung Chun, C. Frank Bennett, Arthur L. Beaudet () and Frank Rigo ()
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Linyan Meng: Baylor College of Medicine, and Texas Children’s Hospital
Amanda J. Ward: Isis Pharmaceuticals
Seung Chun: Isis Pharmaceuticals
C. Frank Bennett: Isis Pharmaceuticals
Arthur L. Beaudet: Baylor College of Medicine, and Texas Children’s Hospital
Frank Rigo: Isis Pharmaceuticals

Nature, 2015, vol. 518, issue 7539, 409-412

Abstract: Angelman syndrome is a neurodevelopmental disorder caused by disrupted function of the maternal copy of the imprinted UBE3A gene; here, targeting a long non-coding RNA that is responsible for silencing the paternal copy of UBE3A with antisense oligonucleotides is shown to partially restore UBE3A expression in the central nervous system and correct some cognitive deficits in a mouse model of the disease.

Date: 2015
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DOI: 10.1038/nature13975

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