公开(公告)号：EP2699673B1

公开(公告)日：2018-09-19

申请号：EP12774899.4

申请日：2012-04-20

申请人： Nationwide Children's Hospital, Inc.

发明人： HARPER, Scott Quenton ,  LIU, Jian

IPC分类号： C12N15/113 ,  A61K48/00 ,  C12N15/864 ,  A61K31/7088 ,  C07K14/47 ,  C12N7/01

CPC分类号： C12N15/86 ,  C07K14/4707 ,  C12N15/113 ,  C12N2310/141 ,  C12N2330/51 ,  C12N2750/14143

摘要： The present invention relates to RNA interference-based methods for inhibiting the expression of the myotilin gene. Recombinant adeno-associated viruses of the invention deliver DNAs encoding microRNAs that knock down the expression of myotilin. The methods have application in the treatment of muscular dystrophies such as Limb Girdle Muscular Dystrophy Type 1A.

公开(公告)号：EP3201337A4

公开(公告)日：2018-06-06

申请号：EP15847837

申请日：2015-10-03

申请人： HOCUSLOCUS LLC ,  UNIV WIEN BODENKULTUR

发明人： EVELETH EDWARD ,  BORTH NICOLE

IPC分类号： C12N15/11 ,  C12N5/10 ,  C12N15/85

CPC分类号： C12N15/111 ,  C12N5/10 ,  C12N15/85 ,  C12N2310/141 ,  C12N2320/10 ,  C12N2330/51 ,  C12N2800/24 ,  C12N2840/102 ,  C12N2840/105 ,  C12N2999/007

摘要： Described herein are methods and compositions for producing a gene of interest (GOD, which, in certain embodiments, can reduce the metabolic burden on cells and reduce decoupling of GOI production from marker production, as compared to prior art methods. The methods relate to positive selection and negative selection approaches to establishing high GOI-producing cell lines, e.g., CHO lines. In certain embodiments, the methods comprise transfecting a cell with (a) an oligonucleotide comprising a GOI and a non-coding RNA, and (b) an oligonucleotide encoding a selection protein; wherein the non-coding RNA promotes or inhibits production of the selection protein. The cell producing the can be identified and/or selected as a result of or by detecting the absence or the presence of the selection protein.

公开(公告)号：EP3290055A3

公开(公告)日：2018-05-30

申请号：EP17181302.5

申请日：2012-07-24

申请人： Nationwide Children's Hospital, Inc.

发明人： HARPER, Scott Quenton ,  LIU, Jian ,  GARWICK, Sara ,  WALLACE, Lindsay

IPC分类号： A61K48/00 ,  C12N15/63 ,  C12N15/09

CPC分类号： C12N15/86 ,  C12N15/113 ,  C12N2310/11 ,  C12N2310/141 ,  C12N2320/30 ,  C12N2330/51 ,  C12N2750/00032 ,  C12N2750/14132 ,  C12N2750/14143

摘要： The present invention relates to RNA interference-based methods for inhibiting the expression of the DUX4 gene, a double homeobox gene on human chromosome 4q35. Recombinant adeno-associated viruses of the invention deliver DNAs encoding microRNAs that knock down the expression of DUX4. The methods have application in the treatment of muscular dystrophies such as facioscapulohumeral muscular dystrophy.

公开(公告)号：EP3202909A4

公开(公告)日：2018-05-23

申请号：EP15847264

申请日：2015-07-23

申请人： RZNOMICS INC

发明人： LEE SEONG WOOK ,  KIM JU HYUN ,  JEONG JIN SOOK ,  HAN SANG YOUNG

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

CPC分类号： A61K31/713 ,  A61K38/00 ,  A61K38/43 ,  C12N15/1135 ,  C12N15/85 ,  C12N15/86 ,  C12N2310/113 ,  C12N2310/124 ,  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.

公开(公告)号：EP3313996A1

公开(公告)日：2018-05-02

申请号：EP16739303.2

申请日：2016-06-24

申请人： Amyris, Inc. ,  Total Marketing Services

发明人： CHUA, Penelope R. ,  JIANG, Hanxiao ,  MEADOWS, Adam Leon

IPC分类号： C12P5/02 ,  C12P7/64 ,  C12P19/62 ,  C12P21/00

CPC分类号： C07K14/395 ,  C07K2319/95 ,  C12N1/16 ,  C12N15/52 ,  C12N15/635 ,  C12N15/65 ,  C12N2330/51 ,  C12P5/02 ,  C12P5/026 ,  C12P7/64 ,  C12P19/62 ,  C12P21/00 ,  C12P21/02 ,  C12Y503/03002

摘要： The present disclosure relates to the use of a maltose dependent degron to control stability of a protein of interest fused thereto at the post-translational level. The present disclosure also relates to the use of a maltose dependent degron in combination with a maltose-responsive promoter to control gene expression at the transcriptional level and to control protein stability at the post-translational level. The present disclosure also relates to the use of a stabilization construct that couples expression of a cell-growth-affecting protein with the production of non-catabolic compounds. The present disclosure further relates to the use of a synthetic maltose-responsive promoter. The present disclosure further provides compositions and methods for using a maltose dependent degron, a maltose-responsive promoter, and a stabilization construct, either alone or in various combinations, for the production of non-catabolic compounds in genetically modified host cells.

公开(公告)号：EP2880167B1

公开(公告)日：2018-04-18

申请号：EP13824780.4

申请日：2013-07-31

申请人： The Board of Regents of The University of Texas System

发明人： DOIRON, Bruno ,  DEFRONZO, Ralph, A.

IPC分类号： C12N15/67

CPC分类号： A61K35/39 ,  A61K31/7088 ,  A61K38/1709 ,  A61K38/45 ,  A61K48/00 ,  C07K14/4702 ,  C12N9/1205 ,  C12N9/16 ,  C12N15/1137 ,  C12N2320/31 ,  C12N2330/51 ,  C12N2740/15043 ,  C12Y207/01002 ,  C12Y301/03048 ,  C12N2310/14 ,  C12N2310/531

摘要： Embodiments of the invention stimulate three levels of beta cell physiology: (i) glucose metabolism, (ii) membrane receptor function, and (iii) transcriptional factors that result in the in vivo formation of beta cells in the pancreas for the purpose of treating diabetes. In certain aspects, the methods include the integration of three levels of cellular physiology: metabolism, membrane receptor function, and gene transcription. The integration of multiple levels of cellular physiology produces a synergistic effect on beta cell formation.

公开(公告)号：EP3137120A4

公开(公告)日：2018-03-14

申请号：EP15785973

申请日：2015-05-01

申请人： UNIV WASHINGTON

发明人： LIEBER ANDRE ,  PAPAYANNOPOULOU THALIA ,  RICHTER MAXIMILIAN ,  SAYDAMINOVA KAMOLA

IPC分类号： A61K48/00

CPC分类号： C12N15/86 ,  A61K35/761 ,  A61K48/0058 ,  A61K48/0091 ,  C12N7/00 ,  C12N15/111 ,  C12N15/113 ,  C12N15/907 ,  C12N2310/141 ,  C12N2330/51 ,  C12N2710/10045 ,  C12N2710/10052 ,  C12N2710/10345 ,  C12N2710/10352 ,  C12N2800/24 ,  C12N2800/50 ,  C12N2800/80 ,  C12N2800/90 ,  C12N2810/6018 ,  C12N2840/007 ,  C12N2840/102 ,  C12N2999/007

摘要： The present invention provides recombinant nucleic acid expression cassetie and helper dependent adenovirus, where the expression cassettes utilize a miRNA based system for controlling expression of nucleases in helper dependent adenoviral viral producer cells, thus permitting production and use for in in vivo gene editing in CD34+ cells.

公开(公告)号：EP3204498A4

公开(公告)日：2018-03-07

申请号：EP15848974

申请日：2015-10-08

申请人： DOW AGROSCIENCES LLC ,  UNIV NEBRASKA

发明人： NARVA KENNETH E ,  ARORA KANIKA ,  WORDEN SARAH ,  RANGASAMY MURUGESAN ,  LI HUARONG ,  FREY MEGHAN ,  SIEGFRIED BLAIR ,  KHAJURIA CHITVAN ,  FISHILEVICH ELANE

IPC分类号： C07K14/325 ,  A01H5/00 ,  A01N57/16 ,  A01N63/02 ,  A01N65/44 ,  C12N5/04 ,  C12N15/113 ,  C12N15/82

CPC分类号： C12N15/8286 ,  A01N57/16 ,  A01N63/02 ,  A01N65/44 ,  C07K14/325 ,  C12N15/113 ,  C12N15/8218 ,  C12N2310/14 ,  C12N2330/51 ,  Y02A40/162 ,  Y02A50/324 ,  A01N65/00

摘要： This disclosure concerns nucleic acid molecules and methods of use thereof for control of insect pests through RNA interference-mediated inhibition of target coding and transcribed non-coding sequences in insect pests, including coleopteran and/or hemipteran pests. The disclosure also concerns methods for making transgenic plants that express nucleic acid molecules useful for the control of insect pests, and the plant cells and plants obtained thereby.

公开(公告)号：EP3191593A4

公开(公告)日：2018-01-24

申请号：EP15840249

申请日：2015-09-08

申请人： UNIV OF IOWA RESEARCH FOUNDATION

发明人： AMENDT BRAD A ,  CAO HUOJUN

IPC分类号： C12N15/113 ,  C12N15/11 ,  C12N15/84 ,  C12N15/86

CPC分类号： C12N15/113 ,  C12N15/111 ,  C12N2310/113 ,  C12N2310/531 ,  C12N2330/51

摘要： The present invention relates to plasmid-based miR inhibitory systems (PMIS), miR inhibitors and methods of use of these systems and inhibitors.

公开(公告)号：EP2530152B1

公开(公告)日：2017-12-27

申请号：EP12176494.8

申请日：2008-05-29

申请人： University of Iowa Research Foundation

发明人： Davidson, Beverly L. ,  Mas Monteys, Alejandro ,  McBride, Jodi L. ,  Boudreau, Ryan

IPC分类号： C12N15/113 ,  A61K31/713

CPC分类号： C12N15/113 ,  C12N15/111 ,  C12N2310/14 ,  C12N2310/141 ,  C12N2310/531 ,  C12N2320/30 ,  C12N2320/53 ,  C12N2330/51

摘要： The present invention is directed to RNA interference (RNAi) molecules targeted against a nucleic acid sequence, and methods of using these RNAi molecules to reduce off-target toxicity.