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1. 发明申请

US20120278913A1 Ribozyme Effector Gene in Dengue Fever Transmission and Disease Control 审中-公开
标题翻译：登革热传播和疾病控制中的核酶效应基因
公开(公告)号：US20120278913A1
公开(公告)日：2012-11-01
申请号：US13096649
申请日：2011-04-28
申请人： Malcolm J. Fraser
发明人： Malcolm J. Fraser
IPC分类号： A01K67/027 , C12N5/10 , C12N15/63
CPC分类号： C12N15/1131 , C12N2310/124 , C12N2510/00 , C12N2770/24122
摘要： Disclosed are anto-DENV ribozyme based methods and compositions useful in the inhibition and control of all Dengue fever serotypes (designated DENV 1 through 4). A group of anti-DENV Group 1 trans-splicing introns (αDENV-GrpIa) are presented that target DENV-2 NGC genomes in situ. Methods for specifically targeting a highly conserved 5′-3′ cyclization sequence (CS) region that is common to all serotypes of the DENV are provided. The anti-DENV Group 1 trans-splicing introns (αDENV-GrpIa) specifically target two different uracil bases on the positive sense genomic strand. The invention provides an RNA based approach for transgeneic suppression of DENV in transformed mosquitoes using a group of specifically designed introns that trans-splice a new RNA sequence downstream of a targeted site. The aDENV-GrpIs target DENV infected genomes and thus provide a method for inhibiting the spread of Dengue fever. An αDENV-GrpI 9v1 is presented that is designed to be active against all forms of Dengue virus, and to effectively target the DENV-2 NGC genome in a sequence specific manner
摘要翻译：公开了基于anto-DENV核酶的方法和组合物，其用于抑制和控制所有登革热血清型（命名为DENV 1至4）。提出了一组抗DENV 1型转拼内含子（αDENV-GrpIa），其原位靶向DENV-2 NGC基因组。提供了特异性靶向DENV所有血清型共有的高度保守的5'-3'环化序列（CS）区域的方法。抗DENV第1组转录内含子（αDENV-GrpIa）特异性靶向阳性基因组链上的两种不同的尿嘧啶碱基。本发明提供了一种基于RNA的方法，用于转化的蚊子中的DENV的转基因抑制方法，其中使用一组专门设计的内含子，其将靶向位点下游的新RNA序列转录。 aDENV-GrpIs靶向DENV感染的基因组，从而提供一种抑制登革热传播的方法。提供了一种被设计为对所有形式的登革热病毒有活性的αDENV-GrpI 9v1，并且以序列特异性方式有效靶向DENV-2 NGC基因组

2. 发明申请

US20090307788A1 RIBOZYME MEDIATED STABILIZATION OF POLYNUCLEOTIDES 审中-公开
标题翻译： RIBOZYME介导的多核苷酸稳定化
公开(公告)号：US20090307788A1
公开(公告)日：2009-12-10
申请号：US12305266
申请日：2007-06-19
申请人： Henrik Nielsen
发明人： Henrik Nielsen
IPC分类号： A01K67/027 , A01H5/00 , C07H21/04 , C07H21/02 , C12N15/63 , C12N5/10 , C12N5/08 , C12P21/06 , A61K35/12 , C12N5/02 , C12N1/21 , C12N1/19 , C12N1/15
CPC分类号： C12N15/111 , C12N9/00 , C12N15/113 , C12N2310/124 , C12N2330/10
摘要： The present invention relates to the production of novel, recombinant polynucleotides comprising the GIR1 ribozyme, or a variant thereof, vectors comprising such polynucleotides and recombinant host cells comprising such polynucleotides and/or such vectors. The invention furthermore relates to the use of said polynucleotides in the treatment of an individual suffering from a disease associated with or caused by instability of a transcript of said second subsequence such as cancer, cachexia, α-Thallasemia or leukaemia.
摘要翻译：本发明涉及包含GIR1核酶或其变体的包含此类多核苷酸的载体和包含此类多核苷酸和/或此类载体的重组宿主细胞的新型重组多核苷酸的生产。本发明还涉及所述多核苷酸在治疗患有与所述第二子序列（例如癌症，恶病质，α-硫代血症或白血病）的转录物不稳定相关或由其引起的疾病的个体中的用途。

3. 发明授权

US06544755B1 Method and reagent for treatment of diseases by expression of the c-Myc gene 失效
标题翻译：通过c-Myc基因表达治疗疾病的方法和试剂
公开(公告)号：US06544755B1
公开(公告)日：2003-04-08
申请号：US08192943
申请日：1994-02-07
申请人： James D. Thompson , Kenneth G. Draper
发明人： James D. Thompson , Kenneth G. Draper
IPC分类号： C07H2104
CPC分类号： C12N15/113 , C12N15/1135 , C12N2310/121 , C12N2310/122 , C12N2310/123 , C12N2310/124 , C12N2310/126
摘要： An enzymatic RNA molecule which cleaves mRNA associated with development or maintenance of Burkitt's lymphoma or acute lymphocytic leukemia.
摘要翻译：一种酶切RNA分子，其切割与发展或维持伯基特氏淋巴瘤或急性淋巴细胞性白血病相关的mRNA。

4. 发明授权

US5872241A Multiple component RNA catalysts and uses thereof 失效
标题翻译：多组分RNA催化剂及其用途
公开(公告)号：US5872241A
公开(公告)日：1999-02-16
申请号：US378235
申请日：1995-01-25
申请人： Anna M. Pyle , William J. Michels
发明人： Anna M. Pyle , William J. Michels
IPC分类号： A01K67/027 , A61K38/00 , C12N15/113 , C07H21/00 , C07H21/02 , C12P19/34 , C12Q1/68
CPC分类号： A01K67/0275 , C12N15/113 , A01K2217/05 , A61K38/00 , C12N2310/111 , C12N2310/124
摘要： This invention is directed to a composition for catalyzed oligonucleotide cleavage comprising a synthetic non-naturally occurring oligonucleotide compound. The compound comprises nucleotides whose sequence defines a conserved group II intron catalytic region and nucleotides whose sequence is capable of hybridizing with a predetermined oligonucleotide target sequence to be cleaved, such target sequence not being present within the compound. The composition also includes an appropriate oligonucleotide co-factor. Preferably, the conserved group II intron catalytic region is a group II intron domain I catalytic region. In one embodiment the conserved group II intron domain I catalytic region may further comprise a conserved portion of a group II intron domain II, a group II intron domain III, a group II intron domain IV, a group II intron domain V, or a group II intron domain VI. The invention is also directed to methods of treatment and methods of use of such compounds.
摘要翻译：本发明涉及包含合成非天然存在的寡核苷酸化合物的催化寡核苷酸切割的组合物。该化合物包含其序列定义保守的第II组内含子催化区域的核苷酸和其序列能够与要切割的预定寡核苷酸靶序列杂交的核苷酸，该目标序列不存在于该化合物内。该组合物还包括合适的寡核苷酸辅因子。优选地，保守的II组内含子催化区是II组内含子结构域I催化区。在一个实施方案中，保守II组内含子结构域I催化区可进一步包含II组内含子结构域II，II组内含子结构域III，II组内含子结构域IV，II组内含子结构域V或组 II内含子结构域VI。本发明还涉及治疗方法和这些化合物的使用方法。

5. 发明授权

US5574143A Ribozymes 失效
公开(公告)号：US5574143A
公开(公告)日：1996-11-12
申请号：US195735
申请日：1994-02-08
申请人： James P. Haseloff , Wayne L. Gerlach , Philip A. Jennings , Fiona H. Cameron
发明人： James P. Haseloff , Wayne L. Gerlach , Philip A. Jennings , Fiona H. Cameron
IPC分类号： A01N57/16 , A01N63/00 , A61K38/00 , C12N15/113 , C12N15/82 , C12N9/10 , A61K38/45 , C12N15/54 , C12N15/64
CPC分类号： C12N15/113 , A01N57/16 , A01N63/00 , C12N15/8216 , C12N15/8218 , A61K38/00 , C12N2310/12 , C12N2310/121 , C12N2310/124
摘要： Compounds having highly specific endoribonuclease activity are described. The compounds of this invention, also known as ribozymes, comprise ribonucleotides having two hybridizing regions with predetermined sequences capable of hybridizing with a target RNA, a region of defined sequence and a base paired stem region.

6. 发明授权

US5543508A Ribozymes 失效
公开(公告)号：US5543508A
公开(公告)日：1996-08-06
申请号：US195072
申请日：1994-02-08
申请人： James P. Haseloff , Wayne L. Gerlach , Philip A. Jennings , Fiona H. Cameron
发明人： James P. Haseloff , Wayne L. Gerlach , Philip A. Jennings , Fiona H. Cameron
IPC分类号： A01N57/16 , A01N63/00 , A61K38/00 , C12N15/113 , C12N15/82 , C12N15/11 , C12N15/70 , C12N15/74 , C12N15/79
CPC分类号： C12N15/113 , A01N57/16 , A01N63/00 , C12N15/8216 , C12N15/8218 , A61K38/00 , C12N2310/12 , C12N2310/121 , C12N2310/124
摘要： Compounds having highly specific endoribonuclease activity are described. The compounds of this invention, also known as ribozymes, comprise nucleotides having two hybridizing regions with predetermined sequences capable of hybridizing with a target RNA, a region of defined sequence and a base paired stem region.

7. 发明授权

US5494814A Ribozymes 失效
标题翻译：核酶
公开(公告)号：US5494814A
公开(公告)日：1996-02-27
申请号：US967693
申请日：1992-10-27
申请人： James P. Haseloff , Wayne L. Gerlach , Philip A. Jennings , Fiona H. Cameron
发明人： James P. Haseloff , Wayne L. Gerlach , Philip A. Jennings , Fiona H. Cameron
IPC分类号： A01N57/16 , A01N63/00 , A61K38/00 , C12N15/113 , C12N15/82 , C12N5/10 , C12N9/22 , C12N15/54 , C12Q1/68
CPC分类号： C12N15/113 , A01N57/16 , A01N63/00 , C12N15/8216 , C12N15/8218 , A61K38/00 , C12N2310/12 , C12N2310/121 , C12N2310/124
摘要： Compounds having highly specific endoribonuclease activity are described. The compounds of this invention, also known as ribozymes, comprise ribonucleotides having two hybridizing regions with predetermined sequences capable of hybridizing with a target RNA, a region of defined sequence and a base paired stem region.
摘要翻译：描述具有高度特异性内切核糖核酸酶活性的化合物。本发明的化合物也称为核酶，其包含具有两个具有能够与靶RNA杂交的预定序列的区域的核糖核苷酸，定义的序列区域和碱基配对的茎区域。

8. 发明申请

US20180187188A1 CANCER SPECIFIC-SPLICING RIBOZYME AND USE THEREOF 审中-公开
公开(公告)号：US20180187188A1
公开(公告)日：2018-07-05
申请号：US15515266
申请日：2015-07-23
申请人： INDUSTRY-ACADEMIC COOPERATION FOUNDATION, DANKOOK UNIVERSITY
发明人： Seong Wook Lee , Ju Hyun Kim , Jin Sook Jeong , Sang Young Han
IPC分类号： C12N15/113 , A61K38/43 , A61K48/00 , C07K14/47 , C12N9/22 , C12N15/79 , A61K31/713 , C12N15/63
CPC分类号： C12N15/113 , A61K31/713 , A61K38/00 , A61K38/43 , A61K48/0058 , A61K48/0066 , C07K14/4705 , C07K2319/01 , C07K2319/33 , C07K2319/61 , C12N9/22 , C12N15/1135 , C12N15/63 , C12N15/79 , C12N15/85 , C12N15/86 , C12N2310/113 , C12N2310/12 , C12N2310/124 , C12N2310/128 , C12N2330/51 , C12N2710/10343 , C12N2830/008 , C12N2830/42 , C12N2830/48
摘要： The present invention relates to a recombination vector, a transformation cell into which the recombinant vector is introduced, a ribozyme expressed from the recombination vector, a prophylactic or therapeutic composition for liver cancer comprising the recombination vector and the ribozyme, and a therapeutic method for liver cancer using the composition, said recombination vector comprising: a tissue-specific promoter; and a ribozyme-target gene expression cassette comprising a trans-splicing ribozyme targeting a cancer-specific gene and a target gene connected to the 3′ exon of the ribozyme, wherein a splicing donor/splicing acceptor sequence (SD/SA sequence) is connected to the 5′ end of the ribozyme-target gene expression cassette, woodchuck hepatitis virus posttranscriptional regulatory element (WPRE) is connected to the 3′ end of the ribozyme-target gene expression cassette, and a nucleic acid sequence recognizing a micro RNA-122a (microRNA-122a, miR-122a) is further connected to the 3′ end of the WPRE.

9. 发明申请

US20180119156A1 RIBOSWITCH INDUCIBLE GENE EXPRESSION 审中-公开
公开(公告)号：US20180119156A1
公开(公告)日：2018-05-03
申请号：US15566346
申请日：2016-04-15
申请人： Wageningen Universiteit
发明人： Sjoerd Constantijn Arnoud CREUTZBURG , John VAN DER OOST
IPC分类号： C12N15/67 , C12N15/70
CPC分类号： C12N15/67 , C12N15/63 , C12N15/70 , C12N2310/124
摘要： An intronic, self-splicing riboswitch is configured for enzyme-product specificity by introducing an appropriate aptamer. This then provides a sensing-expression construct, whereby the presence of an enzyme product in the cell triggers self-splicing of the intron sequence to restore the reading frame of the reporter gene and as such to drive expression of the gene product. The sensing construct expresses a protein which marks the cell or permits its growth or survival in or on an otherwise selective media. In this way, introduction or the presence of such product sensing-reporter constructs in cells can be harnessed to provide a multi-parallel rapid screening of cells or libraries for desirable enzyme variants.

10. 发明申请

US20110256524A1 RECOMBINANT ADENOVIRUS COMPRISING TISSUE-SPECIFIC PROMOTER AND TUMOR-TARGETING TRANS-SPLICING RIBOZYME AND USES THEREOF 审中-公开
标题翻译：包含组织特异性促进剂和肿瘤靶向转移性RIBOZYME的重组腺病毒及其用途
公开(公告)号：US20110256524A1
公开(公告)日：2011-10-20
申请号：US12599671
申请日：2008-08-27
申请人： Seong-Wook Lee , Min-Sun Song , In-Hoo Kim , Jin-Sook Jeong
发明人： Seong-Wook Lee , Min-Sun Song , In-Hoo Kim , Jin-Sook Jeong
IPC分类号： C12Q1/70 , C12N7/01
CPC分类号： C12N15/1135 , A61K38/00 , C12N15/86 , C12N2310/124 , C12N2710/10343 , C12N2830/008 , C12N2840/445
摘要： Disclosed herein are a recombinant adenovirus comprising tissue-specific promoters and trans-splicing ribozymes targeting tumor-specific genes, and uses thereof. More specifically, disclosed herein are a recombinant adenovirus comprising (1) a tissue-specific promoter, (2) a trans-splicing ribozyme acting on tumor-specific genes operably linked to the promoter, and (3) a therapeutic or reporter gene linked to 3′ exon of the ribozyme, an anticancer pharmaceutical composition comprising the same, and a composition for cancer diagnosis comprising the same.The recombinant adenovirus exhibits high specificity and significantly improved therapeutic efficacy to gene targeted tissues. Accordingly, the recombinant adenovirus is useful as a gene delivery vector for anticancer agents or cancer diagnostics.
摘要翻译：本文公开了包含靶向肿瘤特异性基因的组织特异性启动子和反式剪接核酶的重组腺病毒及其用途。更具体地，本文公开了一种重组腺病毒，其包含（1）组织特异性启动子，（2）作用于与启动子可操作地连接的肿瘤特异性基因的反式剪接核酶，和（3）与 3'外显子，包含其的抗癌药物组合物和包含其的用于癌症诊断的组合物。重组腺病毒表现出高特异性，并显着改善了对基因靶向组织的治疗功效。因此，重组腺病毒可用作抗癌剂或癌症诊断的基因递送载体。

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