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1. 发明申请

WO2005007877A2 REGULATABLE PROMOTERS FOR SYNTHESIS OF SMALL HAIRPIN RNA 审中-公开
标题翻译：用于合成小毛发RNA的可调节促进剂
公开(公告)号：WO2005007877A2
公开(公告)日：2005-01-27
申请号：PCT/US2004/023789
申请日：2004-07-19
申请人： UNIVERSITY OF MASSACHUSETTS , XU, Zuoshang , XIA, Xugang
发明人： XU, Zuoshang , XIA, Xugang
IPC分类号： C12Q
CPC分类号： C12N15/111 , A01K2217/058 , C12N2310/111 , C12N2310/14 , C12N2310/53 , C12N2320/50 , C12N2330/30 , C12N2800/30 , C12N2830/003
摘要： The present invention provides compositions for RNA interference and methods of use thereof. The present invention is based on the development of promoters that can be used to regulate shRNA expression spatially (in specific cells) and temporally (at specific times) in cells or transgenic animals that express a recombinase. The compositions and methods of the present invention feature regulatable promoters that allow for inhibition of the expression of target alleles in a spatially and temporally regulatable manner. Thus, the compositions of the present invention are useful for investigating gene functions, both physiologic and pathologic, in specific cell groups and in specific ages, in normal and disease pathways. Functional and genomic and proteomic methods are featured. Therapeutic methods are also featured.
摘要翻译：本发明提供了用于RNA干扰的组合物及其使用方法。本发明是基于开发可以用于在表达重组酶的细胞或转基因动物中空间（特定细胞）和时间（特定时间）调节shRNA表达的启动子的开发。本发明的组合物和方法具有可调节的启动子，其允许以空间和时间上可调节的方式抑制靶基因的表达。因此，本发明的组合物可用于在正常和疾病途径中研究特定细胞组和特定年龄的生理和病理学基因功能。功能和基因组和蛋白质组学方法。还介绍了治疗方法。

2. 发明申请

WO2005096781A3 METHODS AND COMPOSITIONS FOR TREATING GAIN-OF-FUNCTION DISORDERS USING RNA INTERFERENCE 审中-公开
公开(公告)号：WO2005096781A3
公开(公告)日：2005-10-20
申请号：PCT/US2005/011983
申请日：2005-04-06
申请人： UNIVERSITY OF MASSACHUSETTS , XU, Zuoshang , XIA, Xugang
发明人： XU, Zuoshang , XIA, Xugang
IPC分类号： C07H21/04 , A61K31/70
摘要： The present invention relates to novel methods for treating dominant gain-of-function diseases. The invention provides methods for targeting regions of the copper zinc superoxide dismutase (SOD1), which causes inherited amyotrophic lateral sclerosis (ALS), with RNAi agent. The invention further provides RNAi resistant replacement genes containing mismatches with their respective RNAi agent’s. The invention also provides for vectors that express RNAi agent and RNAi resistant replacement gene of the present invention.

3. 发明申请

WO2005007875A2 ENHANCED PROMOTERS FOR SYNTHESIS OF SMALL HAIRPIN RNA 审中-公开
标题翻译：用于合成小毛发RNA的增强促进剂
公开(公告)号：WO2005007875A2
公开(公告)日：2005-01-27
申请号：PCT/US2004/023215
申请日：2004-07-19
申请人： UNIVERSITY OF MASSACHUSETTS , XU, Zuoshang , XIA, Xugang
发明人： XU, Zuoshang , XIA, Xugang
IPC分类号： C12Q
CPC分类号： C12N15/111 , C12N2310/111 , C12N2310/14 , C12N2310/53 , C12N2330/30
摘要： The present invention provides compositions for RNA interference and methods of use thereof. In particular, the invention provides small hairpin RNAs (shRNAs) having modified promoters, including the Pol III U6 promoter, which may be used to increase the potency of shRNA by increasing the expression level. Modifications include constructs with a Pol II enhancer, such as the cytomegalovirus (CMV) enhancer, immediate-early promoter near the Pol III, e.g., U6 promoter, either upstream or downstream from the shRNA sequence and in either forward or backward orientation. Such constructs are useful for increasing the expression of the shRNA, thereby enhancing inhibition of a single nucleotide mismatched mutant allele. Functional and genomic and proteomic methods are featured. Therapeutic methods are also featured.
摘要翻译：本发明提供了用于RNA干扰的组合物及其使用方法。特别地，本发明提供具有修饰的启动子的小发夹RNA（shRNA），其包括Pol III U6启动子，其可用于通过增加表达水平来增加shRNA的效力。修饰包括具有Pol II增强子的构建体，例如巨细胞病毒（CMV）增强子，在Pol III附近的即时早期启动子，例如U6启动子，位于shRNA序列的上游或下游以及向前或向后的方向。这种构建体可用于增加shRNA的表达，从而增强对单核苷酸错配突变体等位基因的抑制。功能和基因组和蛋白质组学方法。还介绍了治疗方法。

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