公开(公告)号：EP2561073A1

公开(公告)日：2013-02-27

申请号：EP11772784.2

申请日：2011-04-22

申请人： University of Massachusetts

发明人： GAO, Guangping ,  ZHANG, Gongwei ,  WANG, Hongyan ,  XU, Zuoshang

IPC分类号： C12N15/00 ,  C12N15/86

CPC分类号： C12N15/86 ,  A61K31/713 ,  A61K38/50 ,  A61K48/00 ,  A61K48/0058 ,  A61K48/0075 ,  C12N7/00 ,  C12N9/80 ,  C12N15/1137 ,  C12N15/635 ,  C12N15/8645 ,  C12N2310/141 ,  C12N2750/14133 ,  C12N2750/14141 ,  C12N2750/14143 ,  C12N2750/14145 ,  C12N2750/14162 ,  C12N2810/10 ,  C12N2840/007 ,  C12Y305/01015

摘要： The invention in some aspects relates to recombinant adeno-associated viruses useful for targeting transgenes to CNS tissue, and compositions comprising the same, and methods of use thereof. In some aspects, the invention provides methods and compositions for treating CNS-related disorders.

公开(公告)号：EP3155101A4

公开(公告)日：2018-04-04

申请号：EP15809732

申请日：2015-06-16

申请人： UNIV JOHNS HOPKINS

发明人： JASKULA RANGA VINOD ,  ZACK DONALD

IPC分类号： C12N15/63 ,  C12N9/16 ,  C12N15/11 ,  C12N15/79 ,  C12N15/85 ,  C12N15/861

CPC分类号： A61K48/0066 ,  C07K2319/10 ,  C07K2319/60 ,  C12N15/111 ,  C12N15/1138 ,  C12N15/63 ,  C12N15/85 ,  C12N15/907 ,  C12N2310/10 ,  C12N2310/20 ,  C12N2330/51 ,  C12N2750/14133 ,  C12N2750/14143 ,  C12N2830/85

摘要： The presently disclosed subject matter provides compositions and methods for the expression of CRISPR guide RNAs using the H1 promoter. In particular, compositions and methods are provided for the use of the H1 promoter to express CRISPR guide RNA (gRNA) with altered specificity of the 5′ nucleotide, as well as use of the H1 promoter sequence as a bidirectional promoter to express Cas9 nuclease and the gRNA simultaneously. Compositions and methods are also provided for the expression and regulation of gRNA expression in vivo through the use of RNA ribozymes and regulatable aptazymes.

公开(公告)号：EP3080143A1

公开(公告)日：2016-10-19

申请号：EP14869305.4

申请日：2014-12-09

申请人： Sangamo BioSciences, Inc.

发明人： MILLER, Jeffrey C. ,  PASCHON, David ,  REBAR, Edward J.

IPC分类号： C07K14/00

CPC分类号： C07K14/47 ,  A61K48/00 ,  C07K14/4702 ,  C07K2319/81 ,  C12N9/22 ,  C12N9/644 ,  C12N15/86 ,  C12N2750/14133 ,  C12N2750/14142 ,  C12N2750/14143

摘要： Disclosed herein are methods and compositions for insertion of transgene sequences encoding proteins involved in clotting into the genome of a cell for treating conditions including hemophilias. In one aspect, described herein is a non-naturally occurring zinc-finger protein (ZFP) that binds to a target site in a region of interest (e.g., an albumin gene) in a genome, wherein the ZFP comprises one or more engineered zinc-finger binding domains. In certain embodiments, the zinc finger domain recognizes a target site in an albumin gene. In some embodiments, the zinc finger protein comprises five or six zinc finger domains designated and ordered F1 to F5 or F1 to F6, as shown in a single row of Table 1.

公开(公告)号：EP3008191A2

公开(公告)日：2016-04-20

申请号：EP14736225.5

申请日：2014-06-12

申请人： Shire Human Genetic Therapies, Inc.

发明人： HEARTLEIN, Michael ,  DEROSA, Frank ,  SMITH, Lianne

IPC分类号： C12N15/86 ,  A61K48/00

CPC分类号： C12N7/00 ,  A61K35/76 ,  A61K38/43 ,  A61K48/00 ,  C12N15/86 ,  C12N2740/10033 ,  C12N2740/10041 ,  C12N2740/10052 ,  C12N2740/15052 ,  C12N2750/14133 ,  C12N2750/14141 ,  C12N2750/14152 ,  C12N2800/40 ,  C12N2999/007

摘要： The present invention provides methods for producing recombinant viral particles based on the use of exogenous mRNAs to supply various helper factors for assembly of viral particles, purified recombinant viral particles produced using such methods, and methods of using such viral particles.

公开(公告)号：EP2673289A4

公开(公告)日：2015-09-23

申请号：EP12744695

申请日：2012-02-10

申请人： UNIV NORTH CAROLINA

发明人： ASOKAN ARAVIND ,  PULICHERLA NAGESH

IPC分类号： C07K14/075 ,  A61P3/00 ,  A61P9/00 ,  A61P21/00 ,  C07K14/005 ,  C12N7/00 ,  C12N15/86 ,  C12N15/861

CPC分类号： C07K14/005 ,  C12N7/00 ,  C12N15/86 ,  C12N2750/14121 ,  C12N2750/14122 ,  C12N2750/14132 ,  C12N2750/14133 ,  C12N2750/14143 ,  C12N2750/14145 ,  C12N2750/14152

公开(公告)号：EP2176283A2

公开(公告)日：2010-04-21

申请号：EP08781733.4

申请日：2008-07-11

申请人： University of Iowa Research Foundation ,  Davidson, Beverly L. ,  Chen, Yong Hong

发明人： DAVIDSON, Beverly, L. ,  CHEN, Yong Hong

IPC分类号： C07K14/025 ,  A61K48/00 ,  C12N15/00 ,  C12N15/34

CPC分类号： A61K38/162 ,  A61K48/00 ,  C07K5/08 ,  C07K5/10 ,  C07K7/06 ,  C07K14/005 ,  C07K2319/33 ,  C12N15/86 ,  C12N2750/14133 ,  C12N2750/14143 ,  C12N2750/14145 ,  C12N2810/40 ,  G01N33/5058

摘要： The present disclosure provides targeting peptides and vectors containing a sequence that encodes targeting peptides that deliver agents to the brain.

公开(公告)号：EP2424991B1

公开(公告)日：2018-06-06

申请号：EP10772363.7

申请日：2010-04-27

申请人： Genzyme Corporation

发明人： PASSINI, Marco, A. ,  SHIHABUDDIN, Lamya ,  CHENG, Seng, H.

IPC分类号： C12N15/864 ,  C07K14/475 ,  C12N15/79 ,  A61K48/00

CPC分类号： A61K38/1709 ,  A61K31/7088 ,  A61K48/00 ,  A61K48/0008 ,  A61K48/005 ,  A61K48/0075 ,  C07K14/4702 ,  C12N7/00 ,  C12N15/86 ,  C12N2750/14121 ,  C12N2750/14133 ,  C12N2750/14143 ,  C12N2750/14171

摘要： Compositions and methods for treating disorders affecting motor function, such as motor function affected by disease or injury to the brain and/or spinal cord, are disclosed.

公开(公告)号：EP3080274A4

公开(公告)日：2017-06-21

申请号：EP14869719

申请日：2014-12-09

申请人： SANGAMO BIOSCIENCES INC

发明人： MILLER JEFFREY C ,  PASCHON DAVID ,  REBAR EDWARD J ,  WECHSLER THOMAS ,  ZHANG LEI

IPC分类号： C07K14/47 ,  A61K48/00 ,  C12N9/22 ,  C12N9/64 ,  C12N15/09 ,  C12N15/62 ,  C12N15/86

CPC分类号： C07K14/47 ,  A61K48/00 ,  C07K14/4702 ,  C07K2319/81 ,  C12N9/22 ,  C12N9/644 ,  C12N15/86 ,  C12N2750/14133 ,  C12N2750/14142 ,  C12N2750/14143

摘要： Disclosed herein are methods and compositions for insertion of transgene sequences encoding proteins involved in clotting into the genome of a cell for treating conditions including hemophilias.

摘要翻译： 本文公开了用于插入转基因序列的方法和组合物，所述转基因序列编码涉及凝结成细胞基因组的蛋白质以治疗包括血友病的病症。

公开(公告)号：EP2561073A4

公开(公告)日：2014-01-08

申请号：EP11772784

申请日：2011-04-22

申请人： UNIV MASSACHUSETTS

发明人： GAO GUANGPING ,  ZHANG HONGWEI ,  WANG HONGYAN ,  XU ZUOSHANG

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

CPC分类号： C12N15/86 ,  A61K31/713 ,  A61K38/50 ,  A61K48/00 ,  A61K48/0058 ,  A61K48/0075 ,  C12N7/00 ,  C12N9/80 ,  C12N15/1137 ,  C12N15/635 ,  C12N15/8645 ,  C12N2310/141 ,  C12N2750/14133 ,  C12N2750/14141 ,  C12N2750/14143 ,  C12N2750/14145 ,  C12N2750/14162 ,  C12N2810/10 ,  C12N2840/007 ,  C12Y305/01015

摘要： The invention in some aspects relates to recombinant adeno-associated viruses useful for targeting transgenes to CNS tissue, and compositions comprising the same, and methods of use thereof. In some aspects, the invention provides methods and compositions for treating CNS-related disorders.

公开(公告)号：EP2673289A1

公开(公告)日：2013-12-18

申请号：EP12744695.3

申请日：2012-02-10

申请人： The University Of North Carolina At Chapel Hill

发明人： ASOKAN, Aravind ,  PULICHERLA, Nagesh

IPC分类号： C07K14/075 ,  C12N15/861 ,  A61P21/00 ,  A61P9/00 ,  A61P3/00

CPC分类号： C07K14/005 ,  C12N7/00 ,  C12N15/86 ,  C12N2750/14121 ,  C12N2750/14122 ,  C12N2750/14132 ,  C12N2750/14133 ,  C12N2750/14143 ,  C12N2750/14145 ,  C12N2750/14152

摘要： The present invention provides AAV capsid proteins, virus capsids comprising said capsid proteins and virus vectors comprising said capsid proteins, wherein the AAV capsid proteins have one or more mutations, wherein the mutation(s) result in a phenotype of decreased liver transduction and/or reduced glycan binding affinity as compared to a control. The invention also provides methods of administering the virus vectors and virus capsids of the invention to a cell or to a subject.