Reversal of phenotypes in MECP2 duplication mice using genetic rescue or antisense oligonucleotides

Yehezkel Sztainberg, Hong-mei Chen, John W. Swann, Shuang Hao, Bin Tang, Zhenyu Wu, Jianrong Tang, Ying-Wooi Wan, Zhandong Liu, Frank Rigo and Huda Y. Zoghbi ()
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Yehezkel Sztainberg: Baylor College of Medicine
Hong-mei Chen: The Cain Foundation Laboratories, Jan and Dan Duncan Neurological Research Institute at Texas Children’s Hospital
John W. Swann: The Cain Foundation Laboratories, Jan and Dan Duncan Neurological Research Institute at Texas Children’s Hospital
Shuang Hao: Jan and Dan Duncan Neurological Research Institute at Texas Children’s Hospital
Bin Tang: Jan and Dan Duncan Neurological Research Institute at Texas Children’s Hospital
Zhenyu Wu: Jan and Dan Duncan Neurological Research Institute at Texas Children’s Hospital
Jianrong Tang: Jan and Dan Duncan Neurological Research Institute at Texas Children’s Hospital
Ying-Wooi Wan: Jan and Dan Duncan Neurological Research Institute at Texas Children’s Hospital
Zhandong Liu: Jan and Dan Duncan Neurological Research Institute at Texas Children’s Hospital
Frank Rigo: Isis Pharmaceuticals
Huda Y. Zoghbi: Baylor College of Medicine

Nature, 2015, vol. 528, issue 7580, 123-126

Abstract: Genetic correction of MeCP2 levels largely reversed the behavioural, molecular and physiological deficits associated with MECP2 duplication syndrome in a transgenic mouse model; similarly, reduction of MeCP2 levels using an antisense oligonucleotide strategy resulted in phenotypic rescue in adult transgenic mice, and dose-dependently corrected MeCP2 levels in cells from patients with MECP2 duplication.

Date: 2015
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DOI: 10.1038/nature16159

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