- 专利标题： Modulation and detection of a neuronal alternative splicing regulatory network for treatment and diagnosis of neurological disorders
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申请号： US15536416申请日： 2015-12-15
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公开(公告)号： US10772928B2公开(公告)日： 2020-09-15
- 发明人: Benjamin J. Blencowe , Mathieu Quesnel-Vallieres , Manuel Irimia , Bushra Raj , Sabine P. Cordes
- 申请人： The Governing Council of the University of Toronto , Sinai Health System
- 申请人地址： CA Toronto, Ontario CA Toronto, Ontario
- 专利权人： The Governing Council of the University of Toronto,Sinai Health System
- 当前专利权人： The Governing Council of the University of Toronto,Sinai Health System
- 当前专利权人地址： CA Toronto, Ontario CA Toronto, Ontario
- 代理机构： Bereskin & Parr LLP/S.E.N.C.R.L., s.r.l.
- 代理商 Ainslie Parsons; Melanie Szweras
- 国际申请： PCT/CA2015/051328 WO 20151215
- 国际公布： WO2016/095037 WO 20160623
- 主分类号： A61K38/55
- IPC分类号： A61K38/55 ; A61K38/05 ; A61K31/7088 ; G01N33/68 ; C12Q1/6883 ; A61K38/46 ; G01N33/50 ; A01K67/027 ; C07K14/47 ; A61K45/06 ; A61K31/713 ; A61K49/00
摘要：
Methods for treatment and diagnosis of neurological disorders such as autism and autism spectrum disorder are disclosed. Also disclosed are modulators of alternative splicing regulators SRRM4 and/or SRRM3 for treating neurological disorders. Further disclosed are agents that modulate the expression of at least one splice variant for treating neurological disorders. Mouse models of neurological disorders having increased or decreased expression of SRRM4 and/or SRRM3 are also disclosed.
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