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Wendy A Gold1,2, Alan K Percy3, Jeffrey L Neul4,5
1Molecular Neurobiology Research Laboratory, Kids Research and Kids Neuroscience Centre, The Children's Hospital at Westmead, School of Medical Sciences, Faculty of Medicine and Health, The University of Sydney, Sydney, New South Wales, Australia.
雷特综合征 (RTT) 是一种严重的神经发育障碍,由MECP2基因突变引起. 对MeCP2缺乏症的研究正在为RTT患者推进诸如托菲尼提德等治疗方法.
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