序号 专利名 申请号 申请日 公开(公告)号 公开(公告)日 发明人
81 METHODS AND COMPOSITIONS FOR THE TARGETED MODIFICATION OF A GENOME EP14790457.7 2014-10-15 EP3080279A1 2016-10-19 FRENDEWEY, David; AUERBACH, Wojtek; LAI, Ka-Man Venus; KUNO, Junko; VALENZUELA, David M.; YANCOPOULOS, George D.
Compositions and methods are provided for modifying a genomic locus of interest in a eukaryotic cell, a mammalian cell, a human cell or a non-human mammalian cell using a large targeting vector (LTVEC) comprising various endogenous or exogenous nucleic acid sequences as described herein. Further methods combine the use of the LTVEC with a CRISPR/Cas system. Compositions and methods for generating a genetically modified non-human animal comprising one or more targeted genetic modifications in their germline are also provided.
82 MESSENGER RNA BASED VIRAL PRODUCTION EP14736225.5 2014-06-12 EP3008191A2 2016-04-20 HEARTLEIN, Michael; DEROSA, Frank; SMITH, Lianne
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.
83 REPRODUCIBLE METHOD FOR TESTIS-MEDIATED GENETIC MODIFICATION (TGM) AND SPERM-MEDIATED GENETIC MODIFICATION (SGM) EP14763005.7 2014-03-17 EP2971006A2 2016-01-20 LANDEL, Carlisle P.; OSTERTAG, Eric M.; RUIZ, Joseph; YESHI, Tseten
The present invention provides a method of direct germline mutagenesis of a non-human animal.
84 CYTOCHROME P450 SUICIDE GENE SYSTEM EP13727093.0 2013-05-28 EP2855681A1 2015-04-08 WIEK, Constanze; KRAMM, Christof; SCHMIDT, Eva
The invention relates to a modified human CYP4B1 protein and a nucleic acid encoding for the modified human CYP4B1 protein. The invention further relates to an expression vector comprising a nucleic acid encoding for a modified human CYP4B1 protein, as well as to a cell comprising a modified CYP4B1 protein. In addition, the invention relates to a modified human CYP4B1 protein for use in treating transplantation induced graft- versus-host-disease (GvHD), and to 4-ipomeanol (4-IPO) for use in killing a cell.
85 PROTEIN HAVING NUCLEASE ACTIVITY, FUSION PROTEINS AND USES THEREOF EP12725813.5 2012-06-06 EP2718440A1 2014-04-16 KÜHN, Ralf
The present invention relates to a nucleic acid molecule encoding (I) a polypeptide having the activity of an endonuclease, which is (a) a nucleic acid molecule encoding a polypeptide comprising or consisting of the amino acid sequence of SEQ ID NO: 1; (b) a nucleic acid molecule comprising or consisting of the nucleotide sequence of SEQ ID NO: 2; (c) a nucleic acid molecule encoding an endonuclease, the amino acid sequence of which is at least 70% identical to the amino acid sequence of SEQ ID NO: 1; (d) a nucleic acid molecule comprising or consisting of a nucleotide sequence which is at least 50% identical to the nucleotide sequence of SEQ ID NO: 2; (e) a nucleic acid molecule which is degenerate with respect to the nucleic acid molecule of (d); or (f) a nucleic acid molecule corresponding to the nucleic acid molecule of any one of (a) to (e) wherein T is replaced by U; (II) a fragment of the polypeptide of (I) having the activity of an endonuclease. Also, the present invention relates to a vector comprising the nucleic acid molecule and a protein encoded by said nucleic acid molecule. Further, the invention relates to a method of modifying the genome of a eukaryotic cell and a method of producing a non-human vertebrate or mammal.
86 METHOD FOR INTRODUCING FOREIGN GENE INTO EARLY EMBRYO OF PRIMATE ANIMAL, AND METHOD FOR PRODUCTION OF TRANSGENIC PRIMATE ANIMAL COMPRISING THE INTRODUCTION METHOD EP08871871.3 2008-12-09 EP2246423A1 2010-11-03 SASAKI, Erika; OKANO, Hideyuki

An object of the present invention is to provide a method for introducing a gene into an embryo for production of a human disease model primate animal using a non-human primate animal such as a marmoset. The present invention relates to a method for introducing a foreign gene into an early embryo of a non-human primate animal, which comprises placing early embryos of a non-human primate in a 0.2 M to 0.3 M sucrose solution, so as to increase the volume of the perivitelline spaces, and then injecting a viral vector containing a human foreign gene operably linked to a promoter into the perivitelline spaces of the early embryos.

87 DIRECTED GENETIC MODIFICATIONS OF HUMAN STEM CELLS PCT/US2004003581 2004-02-06 WO2004072251A3 2004-12-02 ZWAKA THOMAS P; THOMSON JAMES A
Human embryonic stem cells can be genetically transformed by a combination of electroporation and homologous recombination. This technique makes it possible to create targeted inserts or deletions to the genome of the stem cells. This ability makes it possible to create populations of progeny cells which have differentiated into a target cell type of a specific desired lineage.
88 게놈의 표적화된 변형을 위한 방법 및 조성물 KR1020177023915 2014-10-15 KR1020170102056A 2017-09-06 프렌데웨이데이빗; 아워바흐보이테크; 라이카-만베너스; 쿠노준코; 발렌주엘라데이빗엠.; 얀코파울로스죠지디.
본명세서에서설명된바와같이, 다양한내생성또는외생성핵산서열이큰 표적화벡터 (LTVEC)를이용하여, 진핵세포, 포유류세포, 인간세포또는비-인간포유류세포내 관심대상의게놈좌의변형을위한조성물및 방법이제공된다. 추가방법은 LTVEC와 CRISPR/Cas 시스템의이용을복합한다. 생식계통에서하나또는그 이상의표적화된유전적변형이포함된유전적으로변형된비-인간동물을만들기위한조성물및 방법이또한제공된다.
89 재조합 조류 파라믹소바이러스 백신 및 이의 제조 및 사용 방법 KR1020127007373 2010-08-20 KR101745029B1 2017-06-08 부블로미셸; 메바트시온테쇼메; 프리차드조이스; 문트에그베르트
본발명은조작된 APMV 조성물또는백신을포함한다. 백신또는조성물은재조합 APMV 조성물또는백신일수 있다. 본발명은 APMV 의게놈을개질하여재조합 APMV 를제조하는방법; 이방법에의해제조된개질된 APMV; DNA 및단백질서열; 및이 재조합 APMV 로세포및 숙주동물을감염시키는방법을포함한다.
90 유전자 발현 시스템 KR1020177000229 2015-06-04 KR1020170012551A 2017-02-02 코우키도우,마르타; 알페이,루크; 워너,시몬
둘이상의조건부우성치사유전자발현시스템은곤충에서높은수준의침투율을제공한다. 치사는발육의초기단계에서유도되고, 생화학적내성의위험은단일곤충의조건부우선치사유전자발현시스템과비교할때 감소된다. 본발명은곤충개체군의조절에유용하다.
91 게놈의 표적화된 변형을 위한 방법 및 조성물 KR1020167018610 2014-10-15 KR1020160095150A 2016-08-10 프렌데웨이데이빗; 아워바흐보이테크; 라이카-만베너스; 쿠노준코; 발렌주엘라데이빗엠.; 얀코파울로스죠지디.
본명세서에서설명된바와같이, 다양한내생성또는외생성핵산서열이큰 표적화벡터 (LTVEC)를이용하여, 진핵세포, 포유류세포, 인간세포또는비-인간포유류세포내 관심대상의게놈좌의변형을위한조성물및 방법이제공된다. 추가방법은 LTVEC와 CRISPR/Cas 시스템의이용을복합한다. 생식계통에서하나또는그 이상의표적화된유전적변형이포함된유전적으로변형된비-인간동물을만들기위한조성물및 방법이또한제공된다.
92 선택적 마커가 없는 클로닝된 비-인간 동물 KR1020157012263 2013-11-26 KR1020150089004A 2015-08-04 공궈춘; 라이카-만비너스; 발렌주엘라데이비드엠.
ES 세포-특이적프로모터에작동가능하게연결된부위-특이적재조합효소유전자를포함하는자가-절단가능한재조합효소발현카세트를함유하도록유전자조작된, 비-인간동물의유전자변형된체세포가제공된다. 선택적마커유전자및 재조합효소유전자가없는유전자변형및 클로닝된비-인간동물을생산하기위한조성물및 방법이제공되되, ES 세포-특이적프로모터에작동가능하게연결된자가-절단가능한재조합효소유전자를포함하는표적화작제물은분화된체세포내로도입된다. 체세포의유전자변형된게놈은제핵숙주난모세포내로전달된다. 이어서, 인공적으로생성된접합자는배반포배아기까지시험관내에서배양되고, 후속적으로대리모의자궁내로이식되어선택적마커및 재조합효소가없는유전자변형및 클로닝된비-인간동물을형성한다.
93 SYSTEME BACULOVIRAL POUR L'EXPRESSION D'UN VECTEUR DE THERAPIE GENIQUE EP12753763.7 2012-07-27 EP2737072B1 2018-02-14 GALIBERT, Lionel; MERTEN, Otto-Wilhelm; JACOB, Aurélien
94 RECOMBINANT AVIAN PARAMYXOVIRUS VACCINE AND METHOD FOR MAKING AND USING THEREOF EP16204538.9 2010-08-20 EP3210622A2 2017-08-30 BUBLOT, Michel; MEBATSION, Teshome; PRITCHARD, Joyce; MUNDT, Egbert

The present invention encompasses engineered APMV compositions or vaccines. The vaccine or composition may be a recombinant APMV composition or vaccine. The present invention encompasses methods for modifying the genome of APMV to produce recombinant APMV; modified APMV prepared by such methods; DNA and protein sequences; and methods for infecting cells and host animals with such recombinant APMV.

95 GENE EXPRESSION SYSTEM EP15750085.1 2015-06-04 EP3152311A1 2017-04-12 KOUKIDOU, Martha; ALPHEY, Luke; WARNER, Simon
Two or more conditional, dominant, lethal gene expression systems provide high levels of penetrance in insects. Lethality is induced at an earlier stage of development and the risk of biochemical resistance is reduced, as compared to a single insect conditional, dominant, lethal gene expression system. The invention is useful for the control of insect populations.
96 INTRACELLULAR TRANSLATION OF CIRCULAR RNA EP14797117 2014-05-13 EP2996697A4 2017-01-25 KRUSE ROBERT
A circular mRNA molecule possessing features resembling native mammalian mRNA demonstrates improved translation, while retaining the properties of an extremely long half-life inside cells. This circular mRNA is functional inside mammalian cells, being able to compete against native cellular mRNAs for the eukaryotic translation initiation machinery. The invention possesses additional RNA elements compared to a previous invention containing only an IRES element for successful in vitro or in vivo translation.
97 RECOMBINANT AVIAN PARAMYXOVIRUS VACCINE AND METHOD FOR MAKING AND USING THEREOF EP10747133.6 2010-08-20 EP2467158B1 2017-01-25 BUBLOT, Michel; MEBATSION, Teshome; PRITCHARD, Joyce; MUNDT, Egbert
The present invention encompasses engineered APMV compositions or vaccines. The vaccine or composition may be a recombinant APMV composition or vaccine. The present invention encompasses methods for modifying the genome of APMV to produce recombinant APMV; modified APMV prepared by such methods; DNA and protein sequences; and methods for infecting cells and host animals with such recombinant APMV.
98 METHODS FOR DELIVERY TO THE CENTRAL NERVOUS SYSTEM OF NUCLEIC ACID NANOPARTICLES TO TREAT CENTRAL NERVOUS SYSTEM DISORDERS EP13757391 2013-03-11 EP2822600A4 2016-04-06 HARMON BRENDAN; WASCZCAK BARBARA LEE; COOPER MARK
99 GENETICALLY MODIFIED ANIMALS AND METHODS FOR MAKING THE SAME EP13806511 2013-06-19 EP2863736A4 2016-03-09 FAHRENKRUG SCOTT C; CARLSON DANIEL F
Compositions and methods for use of TALENs to make genetically modified livestock or other animals are set forth. Some of the embodiments of the invention provide for making an founder animal that is completely free of all unplanned genetic modifications. Some embodiments are directed to removing genetic faults in established breeds without making other alterations to the genome. Other embodiments are directed to particular tools or processes such as a TALENs with a preferred truncation.
100 CLONED NON-HUMAN ANIMALS FREE OF SELECTIVE MARKERS EP13802213.2 2013-11-26 EP2925122A1 2015-10-07 GONG, Guochun; LAI, Ka-Man, Venus; VALENZUELA, David, M.
Genetically modified somatic cells of a non-human animal are provided that are engineered to contain a self-excisable, recombinase expression cassette comprising a site-specific recombinase gene operably linked to an ES cell-specific promoter. Compositions and methods for producing a genetically modified, cloned non-human animal that is free of a selective marker gene and a recombinase gene are provided, wherein a targeting construct comprising a self-excisable recombinase gene operably linked to an ES cell-specific promoter is introduced into differentiated somatic cells. The genetically modified genome of the somatic cells is transferred into an enucleated host oocyte. The artificially created zygote is then cultured in vitro until the blastocyst embryonic stage and subsequently implanted into a uterus of a surrogate mother to form a genetically modified, cloned non-human animal free of selective marker and recombinase genes.
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