公开(公告)号：EP2350294B1

公开(公告)日：2018-12-05

申请号：EP09752581.0

申请日：2009-10-27

申请人： Baxalta GmbH ,  Baxalta Incorporated

发明人： SCHWARZ, Hans-Peter ,  MUCHITSCH, Eva-Maria ,  TURECEK, Peter

IPC分类号： C12N15/85 ,  A01K67/027 ,  A61K49/00

CPC分类号： A61K49/0008 ,  A01K67/027 ,  A01K67/0275 ,  A01K67/0276 ,  A01K2217/052 ,  A01K2217/054 ,  A01K2217/075 ,  A01K2227/105 ,  A01K2267/03 ,  A01K2267/0306 ,  A01K2267/0325 ,  A01K2267/0381 ,  C12N15/8509

摘要： The invention relates to the development of an animal model for testing various agents in the treatment of a clotting disorder. More specifically, the invention relates to the use of ultra-large molecular weight multimers of von Willebrand factor (VWF) in various mouse strains to induce thrombotic thrombocytopenic purpura (TTP)-like symptoms for the development of a mouse model of TTP. The invention also provides methods for generating such animal disease models and screening methods for identifying biologically active compounds which are effective in the treatment of TTP.

公开(公告)号：EP2844064B1

公开(公告)日：2018-09-05

申请号：EP13730330.1

申请日：2013-05-02

申请人： Istituto Zooprofilattico Sperimentale del Piemonte, Liguria e Valle d'Aosta ,  Avantea S.r.l.

发明人： CASALONE, Cristina ,  GALLI, Cesare ,  CHIEPPA, Maria Novella ,  CORONA, Cristiano ,  BENDOTTI, Caterina ,  PEROTA, Andrea ,  LAZZARI, Giovanna ,  CARAMELLI, Maria ,  LAGUTINA, Irina

IPC分类号： A01K67/027 ,  C12N9/02 ,  C12N15/877 ,  C12N15/85 ,  C12N9/00

CPC分类号： A01K67/0275 ,  A01K2217/052 ,  A01K2227/108 ,  A01K2267/0318 ,  C12N9/0089 ,  C12N15/8509 ,  C12N15/8778 ,  C12N2800/30 ,  C12N2830/46

摘要： Transgenic swine suitable to develop Amyotrophic Lateral Sclerosis, being transgenic for expressing an exogenous gene in neuronal cells, wherein the exogenous gene is a human SOD1 gene carrying a mutation.

公开(公告)号：EP3332008A1

公开(公告)日：2018-06-13

申请号：EP16847694.3

申请日：2016-09-23

申请人： Université Laval

发明人： TREMBLAY, Jacques P. ,  IYOMBE-ENGEMBE, Jean-paul ,  CHAPDELAINE, Pierre

IPC分类号： C12N15/113 ,  A61K31/7088 ,  A61K38/46 ,  A61P3/00 ,  C07H21/02 ,  C12N15/09 ,  C12N15/12 ,  C12N15/55 ,  C12N15/86 ,  C12N5/10 ,  C12N9/22

CPC分类号： C12N15/102 ,  A01K2207/15 ,  A01K2217/052 ,  A01K2227/105 ,  A01K2267/0306 ,  A61K31/7088 ,  A61K31/7105 ,  A61K38/46 ,  A61K45/06 ,  A61K48/005 ,  A61P21/00 ,  C07H21/02 ,  C07K14/4708 ,  C12N9/22 ,  C12N15/11 ,  C12N15/113 ,  C12N15/85 ,  C12N2310/20 ,  A61K2300/00

摘要： Methods of modifying a dystrophin gene are disclosed, for restoring dystrophin expression within a cell having an endogenous frameshift mutation within the dystrophin gene. The methods comprising introducing a first cut within an exon of the dystrophin gene creating a first exon end, wherein said first cut is located upstream of the endogenous frameshift mutation; and introducing a second cut within an exon of the dystrophin gene creating a second exon end, wherein said second cut is located downstream of the frameshift mutation. Upon joining/ligation of said first and second exon ends dystrophin expression is restored, as the correct reading frame is restored. Reagents and uses of the method are also disclosed, for example to treat a subject suffering from muscular dystrophy.

公开(公告)号：EP3327127A1

公开(公告)日：2018-05-30

申请号：EP17199166.4

申请日：2013-12-12

申请人： The Broad Institute, Inc. ,  Massachusetts Institute Of Technology ,  President and Fellows of Harvard College

发明人： Zhang, Feng ,  Heidenreich, Matthias ,  Ran, Fei ,  Swiech, Lukasz

IPC分类号： C12N15/63 ,  C12N15/10

CPC分类号： C12N15/907 ,  A01K67/0275 ,  A01K67/0278 ,  A01K2217/05 ,  A01K2217/052 ,  A01K2217/07 ,  A01K2217/072 ,  A01K2227/105 ,  A01K2267/03 ,  C12N9/22 ,  C12N9/96 ,  C12N15/01 ,  C12N15/102 ,  C12N15/63 ,  C12N15/8213 ,  C12N15/85 ,  C12N15/8509 ,  C12N15/86 ,  C12N15/90 ,  C12N2800/22

摘要： The invention provides for delivery, engineering and optimization of systems, methods, and compositions for manipulation of sequences and/or activities of target sequences. Provided are delivery systems and tissues or organ which are targeted as sites for delivery. Also provided are vectors and vector systems some of which encode one or more components of a CRISPR complex, as well as methods for the design, and use of such vectors. Also provided are methods of directing CRISPR complex formation in eukaryotic cells to ensure enhanced specificity for target recognition and avoidance of toxicity and to edit or modify a, target site in a genomic locus of interest to alter or improve the status of a disease or a condition.

公开(公告)号：EP3262065A1

公开(公告)日：2018-01-03

申请号：EP16756450.9

申请日：2016-02-26

申请人： The Charlotte-Mecklenburg Hospital Authority D/B/A ,  The University of North Carolina at Chapel Hill

发明人： LU, Qi Long ,  XIAO, Xiao

IPC分类号： C07K14/47 ,  C12N15/12 ,  C12N15/861 ,  C12N5/10 ,  C12P21/02 ,  A61K38/17 ,  A61P21/00

CPC分类号： A61K48/0058 ,  A01K67/0275 ,  A01K2217/052 ,  A01K2227/105 ,  A61K38/00 ,  A61K38/1719 ,  C07K14/4707 ,  C12N9/10 ,  C12N15/86 ,  C12N2750/14143 ,  C12N2800/22 ,  C12N2830/008

摘要： The invention relates to synthetic polynucleotides encoding fukutin related protein (FKRP). The invention further relates to nucleic acid constructs comprising the synthetic polynucleotides and methods of using these synthetic polynucleotides to treat dystroglycanopathy disorders.

公开(公告)号：EP3248462A1

公开(公告)日：2017-11-29

申请号：EP17178088.5

申请日：2011-03-31

申请人： Ablexis, LLC

发明人： GREEN, Larry ,  SHIZUYA, Hiroaki

IPC分类号： A01K67/027 ,  C07K16/00 ,  C07K16/18 ,  C07K16/46 ,  C12N15/85

CPC分类号： C07K16/461 ,  A01K67/0275 ,  A01K67/0278 ,  A01K2207/15 ,  A01K2217/052 ,  A01K2217/072 ,  A01K2227/105 ,  A01K2267/01 ,  C07K16/00 ,  C07K16/18 ,  C07K2317/14 ,  C07K2317/24 ,  C07K2317/51 ,  C07K2317/515 ,  C07K2317/52 ,  C07K2317/522 ,  C07K2317/524 ,  C07K2317/526 ,  C07K2317/53 ,  C07K2317/56 ,  C07K2317/64 ,  C12N15/8509 ,  C12N2510/02

摘要： The invention provides a mouse whose genome comprises a transgene comprising a polynucleotide sequence comprising DNA sequences corresponding to DNA sequences from a mouse immunoglobulin locus except for polynucleotide sequences encoding one or more human immunoglobulin heavy chain variable (V) polypeptide sequences, one or more human immunoglobulin heavy chain diversity (D) polypeptide sequences, and one or more human immunoglobulin heavy chain joining (J) polypeptide sequences, wherein the polynucleotide sequences encoding the one or more human heavy chain V polypeptide sequences, the one or more human heavy chain D polypeptide sequences, and the one or more human heavy chain J polypeptide sequences replace one or more mouse immunoglobulin heavy chain V coding sequences, one or more mouse immunoglobulin heavy chain D coding sequences, and one or more mouse immunoglobulin heavy chain J coding sequences, respectively.

摘要翻译： 本发明提供了一种小鼠，其基因组包含含有多核苷酸序列的多核苷酸序列，所述多核苷酸序列包含对应于除编码一个或多个人免疫球蛋白重链可变 重链多肽（D）多肽序列和一个或多个人免疫球蛋白重链连接（J）多肽序列，其中编码一个或多个人重链V多肽序列，一个或多个人重链D多肽序列 并且所述一种或多种人重链J多肽序列分别替代一种或多种小鼠免疫球蛋白重链V编码序列，一种或多种小鼠免疫球蛋白重链D编码序列和一种或多种小鼠免疫球蛋白重链J编码序列。

公开(公告)号：EP3158084A4

公开(公告)日：2017-11-29

申请号：EP15809356

申请日：2015-06-19

申请人： BIOVENTURES LLC

发明人： KIAEI MAHMOUD

IPC分类号： C12Q1/68 ,  A01K67/027 ,  C12N15/85

CPC分类号： C07K14/4716 ,  A01K67/0275 ,  A01K67/0278 ,  A01K2207/15 ,  A01K2217/052 ,  A01K2217/072 ,  A01K2217/206 ,  A01K2227/105 ,  A01K2267/0318 ,  A61K49/0008 ,  C12N15/8509

摘要： The present disclosure provides genetically modified animals and cells comprising a polynucleotide encoding human profilin1. Also provided are methods of assessing the effects of agents in genetically modified animals and cells comprising a polynucleotide encoding human profilin1.

公开(公告)号：EP3003022B1

公开(公告)日：2017-11-22

申请号：EP14817622.5

申请日：2014-06-04

申请人： Virginia Commonwealth University

发明人： FISHER, Paul, B. ,  DAS, Swadesh, K. ,  DASH, Rupesh ,  SARKAR, Devanand ,  SARKAR, Siddik ,  WINDLE, Jolene

IPC分类号： A01K67/027 ,  A61K48/00 ,  C12N15/86

CPC分类号： A61K48/0058 ,  A01K67/0275 ,  A01K2207/12 ,  A01K2217/052 ,  A01K2217/206 ,  A01K2227/105 ,  A01K2267/0331 ,  A01K2267/0393 ,  A61K35/761 ,  A61K49/0008 ,  A61K49/005 ,  C12N15/86 ,  C12N2015/8536 ,  C12N2015/8572 ,  C12N2710/10332 ,  C12N2830/008 ,  C12N2830/34

摘要： Recombinant vectors in which expression of one or more elements (e.g. genes required for viral replication, detectable imaging agents, therapeutic agents, etc.) is driven by a truncated CCN 1 cancer selective promoter (tCCN1-Prom) are provided, as are cells and transgenic animals that contain such vectors. The vectors are used in cancer therapy and/or diagnostics, and the transgenic mice are used to monitor cancer progression, e.g. in screening assays.

公开(公告)号：EP3184632A3

公开(公告)日：2017-10-04

申请号：EP16200960.9

申请日：2010-02-25

申请人： Transposagen Biopharmaceuticals, Inc.

发明人： OSTERTAG, Eric ,  MADISON, Blair

IPC分类号： C12N9/22 ,  A01K67/027 ,  A01K67/033 ,  A61D19/04 ,  A61K48/00 ,  C12N15/85 ,  C12N15/90 ,  C12N9/12

CPC分类号： C12N9/1241 ,  A01K67/0275 ,  A01K67/0333 ,  A01K2217/052 ,  A01K2227/00 ,  A61D19/04 ,  A61K48/0008 ,  C12N9/22 ,  C12N15/8509 ,  C12N15/907 ,  C12N2799/027 ,  C12N2800/90 ,  C12Y207/07

摘要： The present invention provides PiggyBac transposase proteins, nucleic acids encoding the same, compositions comprising the same, kits comprising the same, non-human transgenic animals comprising the same, and methods of using the same.

公开(公告)号：EP3028565B1

公开(公告)日：2017-09-27

申请号：EP16151215.7

申请日：2010-07-07

申请人： Kymab Limited

发明人： BRADLEY, Allan ,  LEE, E-Chiang ,  LIANG, Qi ,  WANG, Wei

IPC分类号： A01K67/027 ,  C07K16/00 ,  C07K16/12 ,  C07K16/18 ,  C07K16/46 ,  C12N15/85 ,  C12N15/90

CPC分类号： A01K67/0278 ,  A01K2207/15 ,  A01K2217/05 ,  A01K2217/052 ,  A01K2217/072 ,  A01K2227/105 ,  A01K2267/01 ,  C07K16/00 ,  C07K16/1239 ,  C07K16/18 ,  C07K16/462 ,  C07K2317/14 ,  C07K2317/21 ,  C07K2317/24 ,  C07K2317/51 ,  C07K2317/56 ,  C07K2317/565 ,  C07K2317/92 ,  C12N15/8509