公开(公告)号：EP0672160A1

公开(公告)日：1995-09-20

申请号：EP94903405.0

申请日：1993-12-02

申请人： TRANSKARYOTIC THERAPIES, INC.

发明人： TRECO, Douglas, A. ,  HEARTLEIN, Michael, W. ,  SELDEN, Richard, F.

IPC分类号： C12N15 ,  A61K31 ,  A61K35 ,  A61K38 ,  A61K48 ,  C07H21 ,  C07K14 ,  C12N5

CPC分类号： C12N15/90 ,  A61K38/00 ,  A61K48/00 ,  C07K14/505 ,  C07K14/61 ,  C07K2319/036 ,  C07K2319/75 ,  C12N15/907

摘要： Method of activating expression of and amplifying an endogenous gene in genomic DNA of a vertebrate cell which is not expressed in the cell as obtained or is not expressed at significant levels in the cell as obtained, comprising the steps of: (a) transfecting cells with DNA sequences comprising: 1) exogenous DNA sequences which repair, alter, delete or replace a sequence present in the cell or which are regulatory sequences not normally functionally linked to the endogenous gene in the cell as obtained; 2) DNA sequences homologous with genomic DNA sequences at a preselected site in the cells; and 3) amplifiable DNA encoding a selectable marker; (b) maintaining the cells under conditions appropriate for homologous recombination to occur between DNA sequences homologous with genomic DNA sequences and genomic DNA sequences; (c) culturing the homologously recombinant cells produced in (b) under conditions which select from amplification of the amplifiable DNA encoding a selectable marker, whereby the amplifiable DNA encoding a selectable marker and the endogenous gene functionally linked to exogenous DNA of (a) 1) are coamplified.

公开(公告)号：EP1503788A2

公开(公告)日：2005-02-09

申请号：EP03726489.2

申请日：2003-04-25

申请人： Transkaryotic Therapies, Inc.

发明人： TRECO, Douglas, A. ,  LOVEDAY, Kenneth ,  BOROWSKI, Marianne

IPC分类号： A61K38/43 ,  A61K38/47

CPC分类号： A61K38/47 ,  A61K38/00 ,  C12Q1/54 ,  C12Y302/01022 ,  G01N2400/10

摘要： The invention provides methods of treating alpha galactosidase A deficiency. Dosage forms, methods of administration, and methods of analyzing human alpha galactosidase A are also included.

公开(公告)号：EP1163349A2

公开(公告)日：2001-12-19

申请号：EP00913825.6

申请日：2000-03-09

申请人： TRANSKARYOTIC THERAPIES, INC.

发明人： SELDEN, Richard, F. ,  BOROWSKI, Marianne ,  KINOSHITA, Carol, M. ,  TRECO, Douglas, A. ,  WILLIAMS, Melanie, D. ,  SCHUETZ, Thomas, J. ,  DANIEL, Peter, F.

IPC分类号： C12N15/56 ,  C12N9/40 ,  C12N15/54 ,  C12N15/63 ,  C12P21/02 ,  A61K38/46

CPC分类号： C07K14/61 ,  A61K38/00 ,  C07K2319/00 ,  C07K2319/02 ,  C12N9/2465 ,  C12Y302/01022

摘要： The invention provides highly purified α-Gal A, and various methods for purifying it; α-Gal A preparations with altered charge and methods for making those preparations; α-Gal A preparations that have an extended circulating half-life in a mammalian host, and methods for making same; and methods and dosages for administering an α-Gal A preparation to a subject.

公开(公告)号：EP0815232A1

公开(公告)日：1998-01-07

申请号：EP96910432.0

申请日：1996-03-12

申请人： TRANSKARYOTIC THERAPIES, INC.

发明人： TRECO, Douglas, A. ,  HEARTLEIN, Michael, W. ,  HAUGE, Brian, M. ,  SELDEN, Richard, F.

IPC分类号： C12N15 ,  A61K35 ,  A61K38 ,  A61K48 ,  A61P7 ,  A61P25 ,  A61P27 ,  A61P37 ,  C07K14 ,  C12N5 ,  C12N9

CPC分类号： A61K48/00 ,  A01K2217/05 ,  A61K38/00 ,  C07K14/505 ,  C07K14/524 ,  C07K14/565 ,  C07K14/605 ,  C07K14/61 ,  C07K2319/00 ,  C07K2319/02 ,  C12N9/16 ,  C12N15/67 ,  C12N15/85 ,  C12N15/907 ,  C12N2510/02 ,  C12N2840/44

摘要： The invention relates to novel human DNA sequences, targeting constructs, and methods for producing novel genes encoding thrombopoietin, DNase I, and β-interferon by homologous recombination. The targeting constructs comprise at least: (a) a targeting sequence; (b) a regulatory sequence; (c) an exon; and (d) a splice-donor site. The targeting constructs, which can undergo homologous recombination with endogenous cellular sequences to generate a novel gene, are introduced into cells to produce homologously recombinant cells. The homologously recombinant cells are then maintained under conditions which will permit transcription of the novel gene and translation of the mRNA produced, resulting in production of either thrombopoietin, DNase I, or β-interferon. The invention further relates to methods of producing pharmaceutically useful preparations containing thrombopoietin, DNase I, or β-interferon from homologously recombinant cells and methods of gene therapy comprising administering homologously recombinant cells producing thrombopoietin, DNase I, or β-interferon to a patient for therapeutic purposes.

公开(公告)号：EP0935651B1

公开(公告)日：2004-12-29

申请号：EP97942567.5

申请日：1997-09-12

申请人： TRANSKARYOTIC THERAPIES, INC.

发明人： SELDEN, Richard, F. ,  BOROWSKI, Marianne ,  GILLESPIE, Frances, P. ,  KINOSHITA, Carol, M. ,  TRECO, Douglas, A. ,  WILLIAMS, Melanie, D.

IPC分类号： C12N9/40 ,  C12N15/62 ,  C12N15/56 ,  A61K38/47 ,  A61K48/00 ,  C12N5/10 ,  C07K14/61

CPC分类号： A61K38/47 ,  A61K48/00 ,  C07K14/61 ,  C07K2319/00 ,  C12N9/2465

摘要： A method of producing human ±-gal A, comprising (a) providing a transfected human cell genetically modified to express human ±-gal A, and (b) obtaining the human ±-gal A from said cultured cells.

公开(公告)号：EP1071768A1

公开(公告)日：2001-01-31

申请号：EP99916683.8

申请日：1999-04-16

申请人： TRANSKARYOTIC THERAPIES, INC.

发明人： LAMSA, Justin, Chase ,  TRECO, Douglas, A.

IPC分类号： C12N15/12 ,  A61K48/00 ,  C12N5/10

CPC分类号： A61K38/37 ,  A61K48/00

摘要： The invention is a method of administering a therapeutic protein to a mammal by introducing into the omentum of the mammal a cell genetically engineered to secrete the therapeutic protein.

公开(公告)号：EP0539490B1

公开(公告)日：1995-11-08

申请号：EP91913696.0

申请日：1991-07-12

申请人： TRANSKARYOTIC THERAPIES, INC.

发明人： TRECO, Douglas, A. ,  MILLER, Allan, M.

IPC分类号： C12Q1/68 ,  C12N15/81 ,  C12Q1/02

CPC分类号： C12N15/1082 ,  C12N15/81 ,  C12N15/905 ,  C12Q1/02 ,  C12Q1/68

摘要： Materials and methods for homologous-recombination screening of DNA libraries constructed in a eukaryotic host and methods for homologous-recombination chromosome walking for isolating overlapping DNA sequences for building an extended physical map of a chromosomal region.

公开(公告)号：EP1071768B1

公开(公告)日：2004-12-29

申请号：EP99916683.8

申请日：1999-04-16

申请人： Transkaryotic Therapies, Inc.

发明人： LAMSA, Justin, Chase ,  TRECO, Douglas, A.

IPC分类号： C12N15/12 ,  A61K48/00 ,  C12N5/10

CPC分类号： A61K38/37 ,  A61K48/00

摘要： The invention is a method of administering a therapeutic protein to a mammal by introducing into the omentum of the mammal a cell genetically engineered to secrete the therapeutic protein.

公开(公告)号：EP1075531A1

公开(公告)日：2001-02-14

申请号：EP99920375.5

申请日：1999-05-05

申请人： TRANSKARYOTIC THERAPIES, INC.

发明人： TRECO, Douglas, A. ,  HEARTLEIN, Michael, W. ,  SELDEN, Richard, F.

IPC分类号： C12N15/67 ,  C12N15/90 ,  C12N15/85 ,  C12N15/21 ,  C07K14/56 ,  A61K48/00 ,  C12N5/10

CPC分类号： C07K14/56 ,  A61K48/00 ,  C12N15/67 ,  C12N15/85 ,  C12N15/907 ,  C12N2840/44

摘要： An isolated nucleic acid molecule that hybridizes under stringent conditions, or shares at least 80 % sequence identity, with a defined genomic region upstream of the coding region of the IFNA2 gene, and a DNA construct containing that DNA molecule as a targeting sequence for homologous recombination.

摘要翻译： 一种分离的核酸分子并在严格条件下杂交，或共有至少80％序列同一性，与IFNA2基因的编码区上游的限定基因组区域，和一个DNA构建体包含做DNA分子作为用于同源重组靶向序列 ，

公开(公告)号：EP1075514A1

公开(公告)日：2001-02-14

申请号：EP99921670.8

申请日：1999-05-05

申请人： TRANSKARYOTIC THERAPIES, INC.

发明人： TRECO, Douglas, A. ,  HEARTLEIN, Michael, W. ,  SELDEN, Richard, F.

IPC分类号： C12N15/09 ,  C12N5/10 ,  C12N15/67 ,  C12N15/85 ,  C07K14/59 ,  A61K48/00

CPC分类号： C07K14/59 ,  A61K48/00

摘要： An isolated nucleic acid molecule that hybridizes under stringent conditions, or shares at least 80 % sequence identity, with a defined genomic region upstream of the coding region of a FSHβ gene, and a DNA construct containing that nucleic acid molecule as a targeting sequence for homologous recombination.