专利快速检索-快速检索全球专利，免费商用专利数据库-IPRDB

1. 发明申请

US20070003963A1 RNA sequence-specific mediators of RNA interference 审中-公开
标题翻译： RNA干扰的RNA序列特异性介质
公开(公告)号：US20070003963A1
公开(公告)日：2007-01-04
申请号：US11474932
申请日：2006-06-26
申请人： Thomas Tuschl , Phillip Zamore , Phillip Sharp , David Bartel
发明人： Thomas Tuschl , Phillip Zamore , Phillip Sharp , David Bartel
IPC分类号： C12Q1/68 , C12N15/86
CPC分类号： C12N15/113 , A01K67/0336 , A01K2207/05 , A01K2217/075 , A01K2227/703 , A01K2267/03 , A61K38/00 , C07H21/02 , C12N15/1079 , C12N15/111 , C12N2310/14 , C12N2310/321 , C12N2310/53 , C12N2330/30 , C12Q1/66 , C12N2310/3521
摘要： The present invention relates to a Drosophila in vitro system which was used to demonstrate that dsRNA is processed to RNA segments 21-23 nucleotides (nt) in length. Furthermore, when these 21-23 nt fragments are purified and added back to Drosophila extracts, they mediate RNA interference in the absence of long dsRNA. Thus, these 21-23 nt fragments are the sequence-specific mediators of RNA degradation. A molecular signal, which may be their specific length, must be present in these 21-23 nt fragments to recruit cellular factors involved in RNAi. This present invention encompasses these 21-23 nt fragments and their use for specifically inactivating gene finction. The use of these fragments (or chemically synthesized oligonucleotides of the same or similar nature) enables the targeting of specific mRNAs for degradation in mammalian cells, where the use of long dsRNAs to elicit RNAi is usually not practical, presumably because of the deleterious effects of the interferon response. This specific targeting of a particular gene function is useful in functional genomic and therapeutic applications.
摘要翻译：本发明涉及果蝇体外系统，其用于证明dsRNA被加工成长度为21-23个核苷酸（nt）的RNA片段。此外，当这些21-23个片段被纯化并加回到果蝇提取物时，它们在不存在长dsRNA的情况下介导RNA干扰。因此，这些21-23个片段是RNA降解的序列特异性介质。必须在这21-23个片段中存在可能是其特异性长度的分子信号，以招募涉及RNAi的细胞因子。本发明包括这些21-23个片段及其用于特异性失活基因功能的用途。使用这些片段（或具有相同或相似性质的化学合成的寡核苷酸）使得能够靶向用于哺乳动物细胞降解的特异性mRNA，其中使用长dsRNA引发RNAi通常是不实际的，可能是因为有害影响干扰素反应。特定基因功能的特异性靶向在功能基因组和治疗应用中是有用的。

2. 发明申请

US20060134787A1 Methods and compositions for enhancing the efficacy and specificity of single and double blunt-ended siRNA 审中-公开
标题翻译：用于增强单端和双端平端siRNA的功效和特异性的方法和组合物
公开(公告)号：US20060134787A1
公开(公告)日：2006-06-22
申请号：US11022055
申请日：2004-12-22
申请人： Phillip Zamore , Dianne Schwarz
发明人： Phillip Zamore , Dianne Schwarz
IPC分类号： A61K48/00 , C12N15/87
CPC分类号： C12N15/113 , C12N15/111 , C12N2310/14 , C12N2320/50 , C12Y115/01001
摘要： The present invention provides methods of enhancing the efficacy and specificity of RNAi using single or double blunt-ended siRNA. The invention also provides single and double-blunt ended siRNA compositions, vectors, and transgenes containing the same for mediating silencing of a target gene. Therapeutic methods are also featured.
摘要翻译：本发明提供使用单或双平端siRNA增强RNAi的功效和特异性的方法。本发明还提供单和双平端的siRNA组合物，载体和含有其的转基因，用于介导靶基因的沉默。还介绍了治疗方法。

3. 发明申请

US20050186586A1 Methods and compositions for enhancing the efficacy and specificity of RNAi 有权
标题翻译：提高RNAi功效和特异性的方法和组合物
公开(公告)号：US20050186586A1
公开(公告)日：2005-08-25
申请号：US10912440
申请日：2004-08-04
申请人： Phillip Zamore , Gyorgy Hutvagner , Dianne Schwarz , Martin Simard
发明人： Phillip Zamore , Gyorgy Hutvagner , Dianne Schwarz , Martin Simard
IPC分类号： A61K48/00 , C12N15/11 , C12N15/113 , C12N15/85 , C12Q1/68
CPC分类号： C12N15/11 , A61K38/00 , A61K48/00 , C12N15/111 , C12N15/113 , C12N2310/111 , C12N2310/14 , C12N2310/331 , C12N2310/333 , C12N2310/336 , C12N2320/51
摘要： The present invention provides methods of enhancing the efficacy and specificity of RNA silencing. The invention also provides compositions for mediating RNA silencing. In particular, the invention provides siRNAs, siRNA-like molecules, shRNAs, vectors and transgenes having improved specificity and efficacy in mediating silencing of a target gene. Therapeutic methods are also featured.
摘要翻译：本发明提供增强RNA沉默的功效和特异性的方法。本发明还提供介导RNA沉默的组合物。特别地，本发明提供了siRNA，siRNA样分子，shRNA，载体和转基因，其具有改善的靶基因沉默的特异性和功效。还介绍了治疗方法。

4. 发明申请

US20050181382A1 Methods and compositions for enhancing the efficacy and specificity of RNAi 有权
标题翻译：提高RNAi功效和特异性的方法和组合物
公开(公告)号：US20050181382A1
公开(公告)日：2005-08-18
申请号：US10859321
申请日：2004-06-02
申请人： Phillip Zamore , Gyorgy Hutvagner , Dianne Schwarz , Martin Simard
发明人： Phillip Zamore , Gyorgy Hutvagner , Dianne Schwarz , Martin Simard
IPC分类号： A61K48/00 , C07H21/02 , C07H21/04 , C12N20060101 , C12N15/11 , C12N15/63 , C12N15/85 , C12N15/88 , C12P19/34 , C12Q1/68
CPC分类号： C12N15/111 , C12N2310/111 , C12N2310/14 , C12N2310/331 , C12N2310/53 , C12N2320/11 , C12N2320/50
摘要： The present invention provides methods of enhancing the efficacy and specificity of RNAi. The invention also provides compositions for mediating RNAi. In particular, the invention provides siRNAs, shRNAs, vectors and transgenes having improved specificity and efficacy in mediating silencing of a target gene. Therapeutic methods are also featured.
摘要翻译：本发明提供增强RNAi功效和特异性的方法。本发明还提供用于介导RNAi的组合物。特别地，本发明提供具有改善的靶基因沉默的特异性和功效的siRNA，shRNA，载体和转基因。还介绍了治疗方法。

5. 发明申请

US20070161591A1 Methods and compositions for treating neurological disease 审中-公开
公开(公告)号：US20070161591A1
公开(公告)日：2007-07-12
申请号：US11506448
申请日：2006-08-18
申请人： Neil Aronin , Phillip Zamore
发明人： Neil Aronin , Phillip Zamore
IPC分类号： A61K48/00 , C07H21/02
CPC分类号： C12N15/113 , A61K31/7088 , A61K47/554 , C12N15/111 , C12N2310/14 , C12N2310/315 , C12N2310/321 , C12N2310/3515 , C12N2320/32 , C12Q1/6883 , C12Q2600/136 , C12Q2600/158 , C12Q2600/178
摘要： This invention relates to methods and compositions for treating neurological disease, and more particularly to methods of delivering iRNA agents to neural cells for the treatment of neurological diseases.

6. 发明申请

US20060009402A1 IN VIVO PRODUCTION OF SMALL INTERFERING RNAS THAT MEDIATE GENE SILENCING 有权
标题翻译：在血液生成小介质基因沉默的小干扰RNA
公开(公告)号：US20060009402A1
公开(公告)日：2006-01-12
申请号：US10195034
申请日：2002-07-12
申请人： Phillip Zamore , Juanita McLachlan , Gyorgy Hutvagner , Alla Grishok , Craig Mello
发明人： Phillip Zamore , Juanita McLachlan , Gyorgy Hutvagner , Alla Grishok , Craig Mello
IPC分类号： A61K48/00
CPC分类号： C12N15/113 , A01K2217/05 , A01K2227/105 , A01K2267/03 , C12N15/111 , C12N15/8509 , C12N2310/111 , C12N2310/14 , C12N2310/53 , C12N2320/30 , C12N2330/30 , C12N2330/51 , C12N2517/02 , C12N2799/021 , C12N2830/008
摘要： The invention provides engineered RNA precursors that when expressed in a cell are processed by the cell to produce targeted small interfering RNAs (siRNAs) that selectively silence targeted genes (by cleaving specific mRNAs) using the cell's own RNA interference (RNAi) pathway. By introducing nucleic acid molecules that encode these engineered RNA precursors into cells in vivo with appropriate regulatory sequences, expression of the engineered RNA precursors can be selectively controlled both temporally and spatially, i.e., at particular times and/or in particular tissues, organs, or cells.
摘要翻译：本发明提供了工程化的RNA前体，其在细胞中表达时被细胞加工以产生靶向小干扰RNA（siRNA），其使用细胞自身的RNA干扰（RNAi）途径选择性沉默靶基因（通过切割特异性mRNA）。通过将具有合适的调节序列将这些工程化RNA前体编码的核酸分子引入细胞体内，工程化RNA前体的表达可以在时间和空间上选择性地控制，即在特定时间和/或特别是组织，器官或细胞。

7. 发明申请

US20050256072A1 Dual functional oligonucleotides for use in repressing mutant gene expression 审中-公开
标题翻译：用于抑制突变基因表达的双功能寡核苷酸
公开(公告)号：US20050256072A1
公开(公告)日：2005-11-17
申请号：US11055035
申请日：2005-02-09
申请人： Neil Aronin , Phillip Zamore , Jennifer Broderick
发明人： Neil Aronin , Phillip Zamore , Jennifer Broderick
IPC分类号： A61K48/00 , C07H21/02 , C12N15/113 , C12N15/63 , C12N15/85 , C12Q1/68
CPC分类号： C12N15/1138 , A61K48/00 , C12N15/113 , C12N15/63 , C12N2310/11 , C12N2310/14 , C12N2310/321 , C12N2310/3519 , C12Q1/6883 , C12Q2600/158 , C12N2310/3521
摘要： The present invention is based, in part, on the discovery that endogenous mRNAs can be recruited for translational repression of target mRNAs. The RNA-silencing agents and the methods described herein, thereby provide a means by which to treat genetic (e.g., genetic neurodegenerative diseases such as Huntington's Disease) or non-genetic diseases by, for example, blocking the synthesis of proteins that contribute to the diseases. Accordingly the RNA-silencing agents of the present invention have an mRNA targeting moiety, a linking moiety, and an mRNA recruiting moiety.
摘要翻译：本发明部分地基于发现内源mRNA可以被募集用于靶mRNA的翻译抑制。 RNA沉默剂和本文所述的方法由此提供了通过例如阻断有助于所述蛋白质的蛋白质的合成来治疗遗传（例如遗传性神经变性疾病例如亨廷顿疾病）或非遗传性疾病的方法疾病因此，本发明的RNA沉默剂具有mRNA靶向部分，连接部分和mRNA募集部分。

8. 发明申请

US20050037988A1 Methods and compositions for controlling efficacy of RNA silencing 有权
标题翻译：用于控制RNA沉默功效的方法和组合物
公开(公告)号：US20050037988A1
公开(公告)日：2005-02-17
申请号：US10859337
申请日：2004-06-02
申请人： Phillip Zamore , Guiliang Tang
发明人： Phillip Zamore , Guiliang Tang
IPC分类号： A61K48/00 , C07H21/00 , C12N20060101 , C12N15/11 , C12N15/82
CPC分类号： C12N15/113 , C12N15/111 , C12N15/8218 , C12N15/8261 , C12N2310/14 , C12N2310/335 , C12N2320/50 , Y02A40/146
摘要： Based at least in part on an understanding of the mechanisms by which small RNAs (e.g., naturally-occurring miRNAs) mediate RNA silencing in plants, rules have been established for determining, for example, the degree of complementarity required between an RNAi-mediating agent and its target, i.e., whether mismatches are tolerated, the number of mismatches tolerated, the effect of the position of the mismatches, etc. Such rules are useful, in particular, in the design of improved RNAi-mediating agents which allow for more exact control of the efficacy of RNA silencing.
摘要翻译：至少部分地基于对小RNA（例如天然存在的miRNA）介导植物中RNA沉默的机制的理解，已经建立了用于确定例如RNAi介导剂之间所需的互补程度的规则以及其目标，即是否容忍不匹配，允许的不匹配的数量，不匹配的位置的影响等。这样的规则特别有用，特别是在改进的RNAi介导试剂的设计中，其允许更准确控制RNA沉默的功效。

9. 发明申请

US20070003962A1 RNA sequence-specific mediators of RNA interference 审中-公开
公开(公告)号：US20070003962A1
公开(公告)日：2007-01-04
申请号：US11474930
申请日：2006-06-26
申请人： Thomas Tuschl , Phillip Zamore , Phillip Sharp , David Bartel
发明人： Thomas Tuschl , Phillip Zamore , Phillip Sharp , David Bartel
IPC分类号： C12Q1/68 , C07H21/02 , C12P19/34
CPC分类号： C12N15/113 , A01K67/0336 , A01K2207/05 , A01K2217/075 , A01K2227/703 , A01K2267/03 , A61K38/00 , C07H21/02 , C12N15/1079 , C12N15/111 , C12N2310/14 , C12N2310/321 , C12N2310/53 , C12N2330/30 , C12Q1/66 , C12N2310/3521
摘要： The present invention relates to a Drosophila in vitro system which was used to demonstrate that dsRNA is processed to RNA segments 21-23 nucleotides (nt) in length. Furthermore, when these 21-23 nt fragments are purified and added back to Drosophila extracts, they mediate RNA interference in the absence of long dsRNA. Thus, these 21-23 nt fragments are the sequence-specific mediators of RNA degradation. A molecular signal, which may be their specific length, must be present in these 21-23 nt fragments to recruit cellular factors involved in RNAi. This present invention encompasses these 21-23 nt fragments and their use for specifically inactivating gene function. The use of these fragments (or chemically synthesized oligonucleotides of the same or similar nature) enables the targeting of specific mRNAs for degradation in mammalian cells, where the use of long dsRNAs to elicit RNAi is usually not practical, presumably because of the deleterious effects of the interferon response. This specific targeting of a particular gene function is useful in functional genomic and therapeutic applications.

10. 发明申请

US20070003961A1 RNA sequence-specific mediators of RNA interference 审中-公开
公开(公告)号：US20070003961A1
公开(公告)日：2007-01-04
申请号：US11474919
申请日：2006-06-26
申请人： Thomas Tuschl , Phillip Zamore , Phillip Sharp , David Bartel
发明人： Thomas Tuschl , Phillip Zamore , Phillip Sharp , David Bartel
IPC分类号： C12Q1/68
CPC分类号： C12N15/113 , A01K67/0336 , A01K2207/05 , A01K2217/075 , A01K2227/703 , A01K2267/03 , A61K38/00 , C07H21/02 , C12N15/1079 , C12N15/111 , C12N2310/14 , C12N2310/321 , C12N2310/53 , C12N2330/30 , C12Q1/66 , C12N2310/3521
摘要： The present invention relates to a Drosophila in vitro system which was used to demonstrate that dsRNA is processed to RNA segments 21-23 nucleotides (nt) in length. Furthermore, when these 21-23 nt fragments are purified and added back to Drosophila extracts, they mediate RNA interference in the absence of long dsRNA. Thus, these 21-23 nt fragments are the sequence-specific mediators of RNA degradation. A molecular signal, which may be their specific length, must be present in these 21-23 nt fragments to recruit cellular factors involved in RNAi. This present invention encompasses these 21-23 nt fragments and their use for specifically inactivating gene function. The use of these fragments (or chemically synthesized oligonucleotides of the same or similar nature) enables the targeting of specific mRNAs for degradation in mammalian cells, where the use of long dsRNAs to elicit RNAi is usually not practical, presumably because of the deleterious effects of the interferon response. This specific targeting of a particular gene function is useful in functional genomic and therapeutic applications.

你已经成功收藏专利！

检索式保存成功!

IPRDB

热门服务

关于我们

友情链接

联系方式