- 专利标题： Methods for treating mendelian disorders of the epigenetic machinery
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申请号： US15315142申请日： 2015-05-29
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公开(公告)号： US11357748B2公开(公告)日： 2022-06-14
- 发明人: Hans Tomas Bjornsson , Harry Dietz
- 申请人： THE JOHNS HOPKINS UNIVERSITY
- 申请人地址： US MD Baltimore
- 专利权人： THE JOHNS HOPKINS UNIVERSITY
- 当前专利权人： THE JOHNS HOPKINS UNIVERSITY
- 当前专利权人地址： US MD Baltimore
- 代理机构： Casimir Jones, S.C.
- 代理商 Tanya A. Arenson
- 国际申请： PCT/US2015/033182 WO 20150529
- 国际公布： WO2015/184260 WO 20151203
- 主分类号： A61K31/20
- IPC分类号： A61K31/20 ; A61K31/167 ; A61K31/12 ; A61K31/55 ; A61K31/60
摘要：
A method is provided for treating a Mendelian disorder of the epigenetic machinery in a subject in need thereof. In particular, the method comprises administering a therapeutically effective amount of an agent that restores balance between open and closed chromatin states at one or more target genes, wherein the agent that restores balance between open and closed chromatin states at one or more target genes is an agent that ameliorates the effect of a defective gene encoding a component of the epigenetic machinery.
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