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REPROGRAMMING-BASED MODELS OF NEURODEVELOPMENTAL DISORDERS AND USES THEREOF

机译：基于重新编程的神经发育障碍模型及其应用

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摘要

The present invention relates to iPSC produced from fibroblast obtained from a subject affected by a neurodevelopmental disorder entailing intellectual disability (ID) and/or a disorder belonging to the Autism Spectrum Disorder (ASD) and/or Schizophrenia (SZ) and uses thereof. The present invention also relates to a cortical neural progenitor cell or a terminally differentiated cortical glutamatergic or gabaergic neuronal cell or a neural crest stem cell line, a mesenchymal stem cell line produced from the iPSC or iPSC line. The invention also relates to method for identifying a compound for the treatment and/or prevention of a neurodevelopmental disorder entailing intellectual disability (ID) and/or a disorder belonging to the Autism Spectrum Disorder (ASD) and/or Schizophrenia (SZ) and to a LSD1 inhibitor or a HDAC2 inhibitor for use in the treatment of such disorders.

机译：本发明涉及由成纤维细胞产生的iPSC及其用途，所述iPSC由得自患有神经发育障碍的受试者的成纤维细胞引起，所述神经发育障碍需要智力障碍（ID）和/或属于自闭症谱系障碍（ASD）和/或精神分裂症（SZ）。本发明还涉及皮层神经祖细胞或终末分化皮层谷氨酸能或加伯能神经元细胞或神经c干细胞系，由iPSC或iPSC系产生的间充质干细胞系。本发明还涉及鉴定用于治疗和/或预防需要智力障碍（ID）和/或属于自闭症谱系障碍（ASD）和/或精神分裂症（SZ）的疾病的神经发育障碍的化合物的方法，并且涉及LSD1抑制剂或HDAC2抑制剂用于治疗此类疾病。

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公开/公告号EP3224352A1

专利类型
公开/公告日2017-10-04

原文格式PDF
申请/专利权人 IEO - ISTITUTO EUROPEO DI ONCOLOGIA SRL;I.R.C.C.S. CASA SOLLIEVO DELLA SOFFERENZA;UNIVERSITA DEGLI STUDI DI MILANO;
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申请/专利号EP20150801762
发明设计人 TESTA GIUSEPPE;ATASHPAZGARGARI SINA;ADAMO ANTONIO;GERMAIN PIERRE-LUC;MERLA GIUSEPPE;DAGOSTINO GIUSEPPE;ZANELLA MATTEO;
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申请日2015-11-25
分类号C12N5/10;C12N5/079;C12Q1/02;A61K31;A61P25;
国家 EP
入库时间 2022-08-21 14:03:22

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