公开(公告)号：US20170335330A1

公开(公告)日：2017-11-23

申请号：US15400698

申请日：2017-01-06

申请人： Shire Human Genetic Therapies, Inc. ,  University of Kentucky

发明人： Stefan Stamm ,  Manli Shen ,  Serene Josiah

IPC分类号： C12N15/113

CPC分类号： C12N15/1138 ,  C12N2310/11 ,  C12N2310/315 ,  C12N2310/321 ,  C12N2310/322 ,  C12N2310/3231 ,  C12N2310/3521 ,  C12N2320/33

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.

公开(公告)号：US20140275222A1

公开(公告)日：2014-09-18

申请号：US14357388

申请日：2012-11-09

申请人： Shire Human Genetic Therapies, Inc. ,  University of Kentucky

发明人： Stefan Stamm ,  Manli Shen ,  Serene Josiah

IPC分类号： C12N15/113

CPC分类号： C12N15/1138 ,  C12N2310/11 ,  C12N2310/315 ,  C12N2310/321 ,  C12N2310/322 ,  C12N2310/3231 ,  C12N2310/3521 ,  C12N2320/33

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.

摘要翻译： 本发明尤其提供了人5-HT 2C受体（HTR2C）的寡核苷酸调节剂以及用于治疗基于这些调节剂的HTR2C相关疾病，病症或病症的改进的方法和组合物。 特别地，根据本发明的寡核苷酸调节剂靶向人HTR2C前mRNA的外显子V /内含子V结中的特异性区域并驱动HTR2C Vb剪接同种型的表达，导致未编辑的强HTR2C受体和增强的5-羟色胺的产生增加 受体活性。

公开(公告)号：US09567585B2

公开(公告)日：2017-02-14

申请号：US14357388

申请日：2012-11-09

申请人： Shire Human Genetic Therapies, Inc. ,  University of Kentucky

发明人： Stefan Stamm ,  Manli Shen ,  Serene Josiah

IPC分类号： C12N15/11 ,  C12N15/113

CPC分类号： C12N15/1138 ,  C12N2310/11 ,  C12N2310/315 ,  C12N2310/321 ,  C12N2310/322 ,  C12N2310/3231 ,  C12N2310/3521 ,  C12N2320/33

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.

摘要翻译： 本发明尤其提供了人5-HT 2C受体（HTR2C）的寡核苷酸调节剂以及用于治疗基于这些调节剂的HTR2C相关疾病，病症或病症的改进的方法和组合物。 特别地，根据本发明的寡核苷酸调节剂靶向人HTR2C前mRNA的外显子V /内含子V结中的特异性区域并驱动HTR2C Vb剪接同种型的表达，导致未编辑的强HTR2C受体和增强的5-羟色胺的产生增加 受体活性。

公开(公告)号：US10415039B2

公开(公告)日：2019-09-17

申请号：US15400698

申请日：2017-01-06

申请人： Shire Human Genetic Therapies, Inc. ,  University of Kentucky

发明人： Stefan Stamm ,  Manli Shen ,  Serene Josiah

IPC分类号： C12N15/11 ,  C12N15/113

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.

公开(公告)号：US20240141349A1

公开(公告)日：2024-05-02

申请号：US18498834

申请日：2023-10-31

申请人： University of Kentucky Research Foundation

发明人： Stefan Stamm ,  Justin Ralph Welden

IPC分类号： C12N15/113

CPC分类号： C12N15/113 ,  C12N2310/11 ,  C12N2310/53

摘要： The present disclosure concerns isolated double stranded (ds) silencing RNA (siRNA) to target a backsplice junction between exons 12 and 7 in the MAPT gene. The ds siRNA include at least one nucleotide from the 3′ terminus of exon 12 linked to at least one nucleotide of the 5′ terminus of exon 7, thereby overlapping the backsplice junction. Targeting the junction ensure that that ds siRNA are limited in targeting the circular RNA produced by the backsplicing while allowing normal expression of the MAPT gene.