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1. 发明申请

WO1994017086A1 GENE REGULATION BY TARGETING PUTATIVE INTRAMOLECULAR TRIPLE HELIX 审中-公开
标题翻译：通过靶向引物分子三肽HELIX进行基因调控
公开(公告)号：WO1994017086A1
公开(公告)日：1994-08-04
申请号：PCT/US1994000348
申请日：1994-01-10
申请人： APOLLON, INC. , YOON, Kyonggeun , LU, Meiqing
发明人： APOLLON, INC.
IPC分类号： C07H17/00
CPC分类号： C12N15/113 , C07H21/00 , C12N15/1131 , C12N15/1132 , C12N15/1133 , C12N15/1135 , C12N15/1136 , C12N15/1138 , C12N2310/152 , C12N2310/315 , C12N2310/321 , C12N2310/53 , C12Q1/6839
摘要： The present invention is related to oligonucleotides or derivatives thereof for decreasing transcription of a target gene having a purine-rich region of DNA that is significantly involved in transcription and has substantial mirror symmetry. The oligonucleotides of the present invention are substantially complementary to the purine stand. Certain preferred embodiments do not form a substantially stable intermolecular triple helix with the target gene in vitro at physiological pH. Other preferred embodiments are oligonucleotides having a circular or stemloop functioning structure that may form both Watson-Crick and Hoogsteen bonds with the target DNA. The invention also provides methods for preparing a composition for decreasing transcription of a target gene, methods for decreasing transcription of a target gene and methods for treating a vertebrate suspecting of having a disease involving the expression of a target gene.
摘要翻译：本发明涉及用于减少具有显着参与转录的具有嘌呤富含DNA区的靶基因的转录的寡核苷酸或其衍生物，并具有实质的镜像对称性。本发明的寡核苷酸与嘌呤体基本上是互补的。某些优选的实施方案在体外在生理pH下不与靶基因形成基本稳定的分子间三螺旋。其它优选的实施方案是具有圆形或茎环功能结构的寡核苷酸，其可以与靶DNA形成沃森 - 克里克和胡格斯汀（Hoogsteen）键。本发明还提供了制备用于降低靶基因转录的组合物的方法，用于降低靶基因转录的方法和用于治疗涉及具有涉及靶基因表达的疾病的脊椎动物的方法。

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