COMPOSITIONS FOR MODULATING MECP2 EXPRESSION专利检索- 其他的一般方法专利检索查询-专利查询网

COMPOSITIONS FOR MODULATING MECP2 EXPRESSION
申请号	US15554409	申请日	2016-03-03	公开(公告)号	US20180036335A1	公开(公告)日	2018-02-08
申请人	Ionis Pharmaceuticals, Inc.;			发明人	Susan M. Freier;
摘要	Disclosed herein are compounds and methods for decreasing methyl CpG binding protein 2 (MECP2) mRNA and protein expression. Such compounds and methods are useful to treat, prevent, or ameliorate MECP2 associated disorders and syndromes. Such MECP2 associated disorders include MECP2 duplication syndrome. In certain embodiments, compounds useful for modulating expression and amount of MECP2 mRNA and protein are antisense compounds. In certain embodiments, the antisense compounds are modified antisense oligonucleotides. In certain embodiments, the antisense compounds are single-stranded antisense oligonucleotides. In certain embodiments, the antisense compounds are not siRNA compounds.
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