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1.发明申请Methods, libraries and computer program products for determining whether siRNA induced phenotypes are due to off-target effects 审中-公开用于确定siRNA诱导表型的方法，文库和计算机程序产品是否由于脱靶效应公开(公告)号：US20080009012A1
公开(公告)日：2008-01-10
申请号：US11825461
申请日：2007-07-06
申请人： Amanda Birmingham , Emily Anderson , Angela Reynolds , Devin Leake , Scott Baskerville , Yuriy Fedorov , Jon Karpilow , William Marshall , Anastasia Khvorova
发明人： Amanda Birmingham , Emily Anderson , Angela Reynolds , Devin Leake , Scott Baskerville , Yuriy Fedorov , Jon Karpilow , William Marshall , Anastasia Khvorova
CPC分类号： C12N15/111 , C12N2310/14 , C12N2320/50 , C12N2330/31
摘要： The present disclosure provides methods, libraries and computer program products for determining whether a phenotype induced by a candidate siRNA for a target gene in an RNAi experiment is target specific or a false positive. Through the use of a control siRNA that has one or two seed sequences of six or seven bases in combination with a neutral scaffolding sequence, a distinction can be made between false positive and true positive analyses of functionality.
摘要翻译： 本公开提供了用于确定由RNAi实验中的靶基因的候选siRNA诱导的表型是靶标特异性还是假阳性的方法，文库和计算机程序产品。 通过使用具有6个或7个碱基的一个或两个种子序列与中性脚手架序列组合的对照siRNA，可以区分功能性的假阳性和真阳性分析。
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2.发明申请Methods, libraries and computer program products for gene silencing with reduced off-target effects 审中-公开用于基因沉默的方法，图书馆和计算机程序产品，减少脱靶效应公开(公告)号：US20070218495A1
公开(公告)日：2007-09-20
申请号：US11724346
申请日：2007-03-15
申请人： Amanda Birmingham , Emily Anderson , Angela Reynolds , Devin Leake , Scott Baskerville , Yuriy Fedorov , Jon Karpilow , William Marshall , Anastasia Khvorova
发明人： Amanda Birmingham , Emily Anderson , Angela Reynolds , Devin Leake , Scott Baskerville , Yuriy Fedorov , Jon Karpilow , William Marshall , Anastasia Khvorova
CPC分类号： C12N15/111 , C12N2310/14 , C12N2320/50 , C12N2330/31
摘要： The present invention provides methods, libraries and computer program products for selecting siRNA that reduce off-target effects and methods for gene silencing using these siRNAs. By comparing nucleotide sequences at positions 2-7 or 2-8 of the sense and/or antisense regions of candidate siRNAs to the 3′ UTR region of mRNAs, one can select siRNAs that have reduced off-target effects.
摘要翻译： 本发明提供用于选择减少脱靶效应的siRNA的方法，文库和计算机程序产品以及使用这些siRNA的基因沉默方法。 通过将候选siRNAs的有义和/或反义区的2-7位或2-8位的核苷酸序列与mRNA的3'UTR区进行比较，可以选择具有降低的脱靶效应的siRNA。
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公开(公告)号：US09353368B2
公开(公告)日：2016-05-31
申请号：US12601508
申请日：2008-05-22
IPC分类号： C12N15/11 , C07H21/04 , C12N15/113 , C12Q1/68
CPC分类号： C12N15/113 , C12N15/111 , C12N2310/141 , C12N2310/531 , C12N2320/30 , C12N2330/30 , C12Q1/6869
摘要： The present disclosure provides a non-naturally occurring miRNA having a stem-loop structure comprising a scaffold derived from a first endogenous miRNA (e.g., miR-196a-2 or miR-204), a mature strand derived from a second endogenous miRNA, and a star strand sequence that is at least partially complementary to the mature strand sequence. The present disclosure also provides a non-naturally occurring miRNA having a stem-loop structure comprising a scaffold derived from an endogenous miRNA (e.g., miR-196a-2 or miR-204), a mature strand designed t be at least partially complementary to a target RNA, and a star strand sequence that is at least partially complementary to the mature strand sequence. The methods and compositions of the disclosure may be used to mediate gene silencing via the RNAi pathway.
摘要翻译： 本公开提供了非天然存在的具有茎环结构的miRNA，其包含源自第一内源miRNA（例如，miR-196a-2或miR-204）的支架，源自第二内源miRNA的成熟链，以及 与成熟链序列至少部分互补的星形链序列。 本公开还提供了非天然存在的miRNA，其具有包含源自内源miRNA（例如，miR-196a-2或miR-204）的支架的茎环结构，成熟链被设计为至少部分地与 靶RNA和与成熟链序列至少部分互补的星形链序列。 本公开的方法和组合物可用于通过RNAi途径介导基因沉默。
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4.发明申请MICRO-RNA SCAFFOLDS, NON-NATURALLY OCCURRING MICRO-RNAS, AND METHODS FOR OPTIMIZING NON-NATURALLY OCCURRING MICRO-RNAS 有权MICRO-RNA SCAFFOLDS，Non-NATURALLY OCCURRING MICRO-RNAS，和METHODS FOR OPTIMIZING NANNA NATURALLY OCCURRING MICRO-RNAS公开(公告)号：US20100256222A1
公开(公告)日：2010-10-07
申请号：US12601506
申请日：2008-05-22
IPC分类号： A61K31/7088 , C07H21/02 , C12N15/63 , C12N5/10 , C12Q1/68
CPC分类号： C12N15/113 , C12N15/111 , C12N2310/141 , C12N2310/531 , C12N2320/30 , C12N2330/30 , C12Q1/6869
摘要： The present disclosure provides non-naturally occurring miR-196a-2 miRNAs and non-naturally occurring miR-204 miRNAs. The non-naturally occurring miRNAs of the disclosure have mature strand sequences distinct from their endogenous counterparts. The disclosure also provides methods of selecting mature strand sequences that function optimally in non-naturally occurring miR-196a-2 miRNAs. The methods and compositions of the disclosure may be used to mediate gene silencing via the RNAi pathway.
摘要翻译： 本公开提供非天然存在的miR-196a-2 miRNA和非天然存在的miR-204 miRNA。 本公开的非天然存在的miRNA具有与其内源对应物不同的成熟链序列。 本公开还提供了选择在非天然存在的miR-196a-2 miRNA中最佳起作用的成熟链序列的方法。 本公开的方法和组合物可用于通过RNAi途径介导基因沉默。
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5.发明申请公开(公告)号：US20100292310A1
公开(公告)日：2010-11-18
申请号：US12601508
申请日：2008-05-22
IPC分类号： A61K31/7088 , C07H21/02 , C12N15/63 , C12N5/10
CPC分类号： C12N15/113 , C12N15/111 , C12N2310/141 , C12N2310/531 , C12N2320/30 , C12N2330/30 , C12Q1/6869
摘要： The present disclosure provides a non-naturally occurring miRNA having a stem-loop structure comprising a scaffold derived from a first endogenous miRNA (e.g., miR-196a-2 or miR-204), a mature strand derived from a second endogenous miRNA, and a star strand sequence that is at least partially complementary to the mature strand sequence. The present disclosure also provides a non-naturally occurring miRNA having a stem-loop structure comprising a scaffold derived from an endogenous miRNA (e.g., miR-196a-2 or miR-204), a mature strand designed t be at least partially complementary to a target RNA, and a star strand sequence that is at least partially complementary to the mature strand sequence. The methods and compositions of the disclosure may be used to mediate gene silencing via the RNAi pathway.
摘要翻译： 本公开提供了非天然存在的具有茎环结构的miRNA，其包含源自第一内源miRNA（例如，miR-196a-2或miR-204）的支架，源自第二内源miRNA的成熟链，以及 与成熟链序列至少部分互补的星形链序列。 本公开还提供了非天然存在的miRNA，其具有包含源自内源miRNA（例如，miR-196a-2或miR-204）的支架的茎环结构，成熟链被设计为至少部分地与 靶RNA和与成熟链序列至少部分互补的星形链序列。 本公开的方法和组合物可用于通过RNAi途径介导基因沉默。
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6.发明授权Micro-RNA scaffolds, non-naturally occurring micro-RNAs, and methods for optimizing non-naturally occurring micro-RNAs 有权微RNA支架，非天然存在的微RNA，以及优化非天然存在的微RNA的方法公开(公告)号：US08841267B2
公开(公告)日：2014-09-23
申请号：US12601506
申请日：2008-05-22
CPC分类号： C12N15/113 , C12N15/111 , C12N2310/141 , C12N2310/531 , C12N2320/30 , C12N2330/30 , C12Q1/6869
摘要： The present disclosure provides non-naturally occurring miR-196a-2 miRNAs and non-naturally occurring miR-204 miRNAs. The non-naturally occurring miRNAs of the disclosure have mature strand sequences distinct from their endogenous counterparts. The disclosure also provides methods of selecting mature strand sequences that function optimally in non-naturally occurring miR-196a-2 miRNAs. The methods and compositions of the disclosure may be used to mediate gene silencing via the RNAi pathway.
摘要翻译： 本公开提供非天然存在的miR-196a-2 miRNA和非天然存在的miR-204 miRNA。 本公开的非天然存在的miRNA具有与其内源对应物不同的成熟链序列。 本公开还提供了选择在非天然存在的miR-196a-2 miRNA中最佳起作用的成熟链序列的方法。 本公开的方法和组合物可用于通过RNAi途径介导基因沉默。
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