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

Author

Listed:
  • 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)

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.

Suggested Citation

  • Linyan Meng & Amanda J. Ward & Seung Chun & C. Frank Bennett & Arthur L. Beaudet & Frank Rigo, 2015. "Towards a therapy for Angelman syndrome by targeting a long non-coding RNA," Nature, Nature, vol. 518(7539), pages 409-412, February.
    • Handle: RePEc:nat:nature:v:518:y:2015:i:7539:d:10.1038_nature13975
    DOI: 10.1038/nature13975
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