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THERAPIES FOR THE TREATMENT OF DISEASES AND DISORDERS ASSOCIATED WITH ABNORMAL EXPRESSION OF A NEURAL-ASSOCIATED GENE
THERAPIES FOR THE TREATMENT OF DISEASES AND DISORDERS ASSOCIATED WITH ABNORMAL EXPRESSION OF A NEURAL-ASSOCIATED GENE
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机译:治疗与神经相关基因异常表达相关的疾病和疾病的疗法
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摘要
The disclosure provides methods for rescuing synaptic defects caused by abnormal MeCP2 expression in a subject in need thereof, comprising administering to the subject therapeutically effective amount(s) of a nootropic agent and/or an alpha-7 nicotinic acetylcholine receptor (α7-nAChR) agonist. The disclosure further provides methods for screening candidate drug candidates in a tiered series of assays and models (neurons, MECP2-mosaic neurospheres, and cortical organoids).
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