发明名称 |
ANTISENSE OLIGONUCLEOTIDE MODULATORS OF SEROTONIN RECEPTOR 2C AND USES THEREOF |
摘要 |
The present invention provides, among other things, oligonucleotide modulators of human 5'-HT2C receptor (HTR2C) and improved methods and composition for treating HTR2C-related diseases, disorders or conditions based on such modulators. In particular, oligonucleotides modulators according to the invention target specific regions in the Exon V/Intron V junction of the human HTR2C pre-mRNA and drive expression of HTR2C Vb splice isoform, leading to increased generation of non-edited strong HTR2C receptor and enhanced serotonin receptor activity. |
申请公布号 |
WO2013071022(A1) |
申请公布日期 |
2013.05.16 |
申请号 |
WO2012US64319 |
申请日期 |
2012.11.09 |
申请人 |
SHIRE HUMAN GENETIC THERAPIES, INC.;UNIVERSITY OF KENTUCKY |
发明人 |
STAMM, STEFAN;SHEN, MANLI;JOSIAH, SERENE |
分类号 |
C07H21/00;C12N15/113 |
主分类号 |
C07H21/00 |
代理机构 |
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代理人 |
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主权项 |
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地址 |
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