公开(公告)号：EP3146051A4

公开(公告)日：2017-12-20

申请号：EP15795373

申请日：2015-05-20

申请人： UNIV OF IOWA RESEARCH FOUNDATION

发明人： DAVIDSON BEVERLY L ,  MAS MONTEYS ALEJANDRO

IPC分类号： C12N15/113 ,  A61K31/7088

CPC分类号： C12N15/113 ,  A61K9/0085 ,  A61K31/7088 ,  C12N2310/14 ,  C12N2310/141 ,  C12N2320/30 ,  C12N2320/32 ,  C12N2330/51

摘要： The present invention is directed to RNA interference (RNAi) molecules targeted against a Huntington's disease nucleic acid sequence, and methods of using these RNAi molecules to treat Huntington's disease.

公开(公告)号：EP3204504A1

公开(公告)日：2017-08-16

申请号：EP15848994.8

申请日：2015-10-09

申请人： Dana-Farber Cancer Institute, Inc.

发明人： KAELIN, William ,  LU, Gang ,  MIDDLETON, Richard ,  WONG, Kwok Kin ,  BRADNER, James E.

IPC分类号： C12N15/86 ,  C12N15/63 ,  C40B40/02

CPC分类号： G01N33/6845 ,  C12N15/1082 ,  C12N15/113 ,  C12N15/1137 ,  C12N15/64 ,  C12N2310/10 ,  C12N2310/14 ,  C12N2310/20 ,  C12N2330/51 ,  G01N2500/10 ,  C12N2310/531

摘要： The present application relates, in some aspects, to the development of a plasmid that can be used to efficiently monitor the stabilities of thousands of proteins after specific perturbations. The plasmid allows for the co-expression of two reporter proteins, each of which is placed under the control of an IRES. Other aspects of the invention relate to a plasmid library, screening methods to identify proteins whose levels are modulated by a compound of interest, and methods for monitoring treatment of a subject with an IMiD compound.

摘要翻译： 在一些方面，本申请涉及开发可用于在特定扰动之后有效监测数千种蛋白质的稳定性的质粒。 该质粒允许两种报道蛋白的共表达，每种报道蛋白被置于IRES的控制之下。 本发明的其它方面涉及质粒文库，鉴定其水平受感兴趣化合物调节的蛋白质的筛选方法，以及用IMiD化合物监测对象治疗的方法。

公开(公告)号：EP3202909A1

公开(公告)日：2017-08-09

申请号：EP15847264.7

申请日：2015-07-23

申请人： Industry-Academic Cooperation Foundation Dankook University

发明人： LEE, Seong Wook ,  KIM, Ju Hyun ,  JEONG, Jin Sook ,  HAN, Sang Young

IPC分类号： C12N15/85 ,  C12N9/22 ,  A61K31/713 ,  A61K38/43 ,  A61P35/00

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.

摘要翻译： 本发明涉及重组载体，导入重组载体的转化细胞，由该重组载体表达的核酶，含有重组载体和核酶的肝癌的预防或治疗组合物，以及肝的治疗方法 使用所述组合物的癌症，所述重组载体包含：组织特异性启动子; 和包含靶向癌症特异性基因的转拼核酶和连接于核酶的3'外显子的靶基因的核酶靶基因表达盒，其中连接剪接供体/剪接受体序列（SD / SA序列） 到核酶靶基因表达盒的5'末端，将土拨鼠肝炎病毒转录后调节元件（WPRE）连接到核酶靶基因表达盒的3'末端，并且将识别微小RNA-122a的核酸序列 （microRNA-122a，miR-122a）进一步连接到WPRE的3'末端。

公开(公告)号：EP3155128A2

公开(公告)日：2017-04-19

申请号：EP15732984.8

申请日：2015-06-10

申请人： Erasmus University Medical Center Rotterdam

发明人： BERGSMA, Atze Jacobus ,  VAN DER WAL, Erik ,  PIJNAPPEL, Wilhelmus Wenceslaus Matthias ,  VAN DER PLOEG, Antje Tjitske ,  REUSER, Arnoldus

IPC分类号： C12Q1/68 ,  A61K48/00 ,  C12N15/11

CPC分类号： C12Q1/6876 ,  C12N15/111 ,  C12N15/113 ,  C12N2310/11 ,  C12N2320/33 ,  C12N2330/30 ,  C12N2330/50 ,  C12N2330/51 ,  C12Q1/6846 ,  C12Q1/6851 ,  C12Q1/6883 ,  C12Q2600/136 ,  C12Q2600/156

摘要： The disclosure provides method and kits for characterizing spliced m RNA isoforms. The disclosure also provides methods of screening for mutations and oligonucleotides that modulate splicing.

摘要翻译： 本公开提供用于表征剪接的m RNA亚型的方法和试剂盒。 本公开还提供筛选调节剪接的突变和寡核苷酸的方法。

公开(公告)号：EP3149170A1

公开(公告)日：2017-04-05

申请号：EP15729007.3

申请日：2015-05-29

申请人： The Board of Trustees of the Leland Stanford Junior University

发明人： QUAKE, Stephen R. ,  WANG, Jianbin

IPC分类号： C12N15/113 ,  A61K31/7088 ,  A61P31/22

CPC分类号： C12N15/907 ,  A61K38/00 ,  A61K48/005 ,  C12N9/16 ,  C12N9/22 ,  C12N15/1133 ,  C12N15/86 ,  C12N2310/20 ,  C12N2330/51 ,  C12N2810/60 ,  C12N2820/60 ,  C12Y301/00 ,  Y02A50/385 ,  Y02A50/387 ,  Y02A50/393 ,  Y02A50/463 ,  Y02A50/465 ,  Y02A50/467

摘要： Viral infection is a persistent cause of human disease. Guided nuclease systems target the genomes of viral infections, rendering the viruses incapacitated.

摘要翻译： 病毒感染是人类疾病的持续原因。 引导核酸酶系统针对病毒感染的基因组，使病毒丧失能力。

公开(公告)号：EP2924115A4

公开(公告)日：2016-07-13

申请号：EP13857052

申请日：2013-11-21

申请人： NAT CANCER CT

发明人： LEE SANG-JIN ,  KIM YUN-HEE ,  KIM IN-HOO ,  LEE SEONG-WOOK ,  JEONG JIN-SOOK ,  HAN SANG YOUNG

IPC分类号： C12N7/01 ,  A61K35/76 ,  C12Q1/02

CPC分类号： C12N7/00 ,  A61K31/713 ,  A61K35/761 ,  A61K38/45 ,  A61K48/0066 ,  C12N15/1135 ,  C12N15/86 ,  C12N2310/1241 ,  C12N2320/32 ,  C12N2330/51 ,  C12N2710/10021 ,  C12N2710/10032 ,  C12N2710/10343 ,  C12N2710/16633 ,  C12N2810/6018 ,  C12N2830/008 ,  C12N2830/42 ,  C12N2840/445 ,  C12Y207/01021 ,  C12Y207/07049 ,  G01N33/56983 ,  G01N33/57438 ,  G01N2333/075

摘要： The present invention relates to a recombinant adenovirus with increased in-vivo safety, tissue specificity, and anticancer activities, and a use thereof. Specifically, the recombinant adenovirus comprising: a promoter of the liver tissue-specific phosphoenolpyruvate carboxykinase (PEPCK) gene; a trans-splicing ribozyme which is operably linked to the promoter and acts on a cancer-specific gene; a therapeutic gene or a reporter gene which is linked to the 3' exon of the ribozyme; and a serotype 35 fiber knob and a serotype 5 shaft, in which the orf4 gene is deleted from adenovirus E1, E3 and E4 orf1, shows remarkable in-vivo safety, high specificity for a target tissue, and remarkable anticancer effects, and thus can be useful for an anticancer drug or a cancer diagnostic agent as a gene delivery vector.

公开(公告)号：EP2764123A4

公开(公告)日：2015-07-01

申请号：EP12839041

申请日：2012-10-02

申请人： GE HEALTHCARE DHARMACON INC

发明人： WAKEFIELD JOHN

IPC分类号： C12Q1/68 ,  C12N15/00 ,  C12N15/113

CPC分类号： C12N15/1086 ,  C12N15/111 ,  C12N2330/51 ,  C12N2740/16043

摘要： Lentiviral vectors, plates, kits and methods are provided that permit improved efficiency for selection of a promoter sequence for use in a lentiviral application. Through various embodiments of the present invention, a researcher may first evaluate and then choose in a modular fashion, a vector comprising a promoter that is effective for expression in a particular cell line or type.

公开(公告)号：EP2885409A1

公开(公告)日：2015-06-24

申请号：EP13753070.5

申请日：2013-08-16

申请人： Novozymes A/S

发明人： BRODY, Howard ,  RICACHO, Evee ,  TERAMOTO, Hiroshi

IPC分类号： C12N15/113 ,  C12N15/80 ,  C12P21/00

CPC分类号： C12N15/80 ,  C12N15/1086 ,  C12N15/113 ,  C12N2310/14 ,  C12N2310/53 ,  C12N2320/12 ,  C12N2330/51

摘要： The present invention relates to methods for co-silencing expression of genes in a filamentous fungal strain by transitive RNA interference. The present invention also relates to methods for identifying a gene encoding a biological substance of interest.

公开(公告)号：EP2871239A9

公开(公告)日：2015-06-24

申请号：EP13813664.3

申请日：2013-06-26

申请人： Takara Bio, Inc.

发明人： NISHIE, Toshikazu ,  TAKASHIMA, Fuyuko ,  ENOKI, Tatsuji ,  MINENO, Junichi

IPC分类号： C12N15/113 ,  C12N5/10 ,  C12N15/09

CPC分类号： C12N15/86 ,  C12N5/10 ,  C12N7/00 ,  C12N15/09 ,  C12N15/111 ,  C12N15/64 ,  C12N2310/141 ,  C12N2330/51 ,  C12N2501/65 ,  C12N2750/14143 ,  C12N2750/14151 ,  C12P19/34

摘要： According to the present invention, an AAV vector having a higher titer compared with those of conventional ones can be produced using a cell into which a nucleic acid capable of expressing miRNA is introduced artificially. An AAV vector produced using the cell and a composition containing the viral vector as an active ingredient are very useful as gene transfer means in the studies or clinical practice of gene therapies.

公开(公告)号：EP2871239A1

公开(公告)日：2015-05-13

申请号：EP13813664.3

申请日：2013-06-26

申请人： Takara Bio, Inc.

发明人： NISHIE, Toshikazu ,  TAKASHIMA, Fuyuko ,  ENOKI, Tatsuji ,  MINENO, Junichi

IPC分类号： C12N15/113 ,  C12N5/10 ,  C12N15/09

CPC分类号： C12N15/86 ,  C12N5/10 ,  C12N7/00 ,  C12N15/09 ,  C12N15/111 ,  C12N15/64 ,  C12N2310/141 ,  C12N2330/51 ,  C12N2501/65 ,  C12N2750/14143 ,  C12N2750/14151 ,  C12P19/34

摘要： According to the present invention, an AAV vector having a higher titer compared with those of conventional ones can be produced using a cell into which a nucleic acid capable of expressing miRNA is introduced artificially. An AAV vector produced using the cell and a composition containing the viral vector as an active ingredient are very useful as gene transfer means in the studies or clinical practice of gene therapies.

摘要翻译： 根据本发明，可以使用其中人工引入了能够表达miRNA的核酸的细胞来生产与常规的AAV载体相比具有更高滴度的AAV载体。 使用该细胞和含有病毒载体作为活性成分的组合物产生的AAV载体作为基因治疗的研究或临床实践中的基因转移手段非常有用。