公开(公告)号：EP3393522A1

公开(公告)日：2018-10-31

申请号：EP16825400.1

申请日：2016-12-21

申请人： INSERM (Institut National de la Santé et de la Recherche Médicale) ,  Université de Nantes ,  Centre Hospitalier Universitaire De Nantes ,  Université de Montpellier

发明人： KALATZIS, Vasiliki ,  FRANCOIS, Achille

IPC分类号： A61K48/00 ,  C07K14/705 ,  C12N15/86

CPC分类号： C12N15/86 ,  A61K48/005 ,  C12N2750/14143 ,  C12N2800/40 ,  C12N2840/20 ,  C12N2840/445

摘要： The invention relates to constructs, vectors, relative host cells and pharmaceutical compositions which allow an effective gene therapy, in particular of genes larger than 5Kb by using an improved hybrid dual recombinant AAV vector system.

公开(公告)号：EP2320952A1

公开(公告)日：2011-05-18

申请号：EP09802455.7

申请日：2009-07-30

申请人： Bauer, Johann

发明人： MITCHELL, Lloyd, G.

IPC分类号： A61K48/00

CPC分类号： C12N15/85 ,  A61K48/005 ,  C07K14/78 ,  C12N2740/13043 ,  C12N2840/445

摘要： The present invention relates to specific and markedly improved pre-mRNA
trans -splicing molecule (RTM) molecules which are designed to correct specific genes expressed within cells to be targeted, and which are associated with epidermolysis bullosa, cystic fibrosis, pachyonychia congenital, and psoriasis or neurodermitis, as well as cancers of the skin. In particular, the RTMs of the present invention are genetically engineered to interact with a specific target pre-mRNA expressed in cells to be targeted so as to result in correction of genetic defects or reprogramming of gene expression responsible for a variety of different skin disorders.

摘要翻译： 本发明涉及特异性和显着改进的mRNA前转录分子（RTM）分子，其被设计用于校正在靶细胞内表达的特异性基因，并且其与大疱性表皮松解，囊性纤维化，先天性先天性和牛皮癣相关 或神经性皮炎，以及皮肤癌。 特别地，本发明的RTM被遗传工程化以与要靶向的细胞中表达的特异性靶mRNA前体相互作用，从而导致基因缺陷的校正或负责多种不同皮肤病症的基因表达的重新编程。

公开(公告)号：EP0883344B1

公开(公告)日：2007-02-28

申请号：EP96944760.6

申请日：1996-12-13

申请人： Intronn, Inc.

发明人： MITCHELL, Lloyd, G.

IPC分类号： A01N43/04 ,  A61K31/70 ,  C07H21/02 ,  C07H21/04 ,  C12N15/00 ,  C12N15/09 ,  C12N15/63 ,  C12N15/70 ,  C12N15/74

CPC分类号： A61K48/005 ,  A61K38/00 ,  A61K48/00 ,  C07K14/34 ,  C12N15/1027 ,  C12N15/111 ,  C12N15/63 ,  C12N15/85 ,  C12N2320/33 ,  C12N2840/44 ,  C12N2840/445

公开(公告)号：EP1358203A4

公开(公告)日：2006-11-22

申请号：EP02714709

申请日：2002-01-08

申请人： INTRONN INC

发明人： MITCHELL LLOYD G ,  GARCIA-BLANCO MARIANO A ,  BAKER CARL C ,  PUTTARAJU MADAIAH ,  MANSFIELD GARY S ,  CHAO HENGJUN

IPC分类号： C12N15/09 ,  C12N15/85 ,  A61K35/76 ,  A61K38/43 ,  A61K48/00 ,  A61P31/00 ,  A61P31/04 ,  A61P35/00 ,  A61P37/02 ,  A61P37/06 ,  C12N1/15 ,  C12N1/19 ,  C12N1/21 ,  C12N5/10 ,  C12N15/10

CPC分类号： C12N15/85 ,  C12N15/1027 ,  C12N2800/108 ,  C12N2830/001 ,  C12N2830/60 ,  C12N2840/44 ,  C12N2840/445

公开(公告)号：EP1555874A4

公开(公告)日：2006-10-04

申请号：EP03774728

申请日：2003-10-09

申请人： OXFORD BIOMEDICA LTD

发明人： JOLLY DOUG ,  ROHLL JONATHAN ,  BUEHLER BERND ,  MITROPHANOUS KYRI ,  RADCLIFF PIPPA

IPC分类号： A61K48/00 ,  C12N15/63 ,  C12N15/67 ,  C12N15/86 ,  C12N15/867 ,  C12Q1/68

CPC分类号： C12N15/67 ,  C12N15/63 ,  C12N15/86 ,  C12N2740/15043 ,  C12N2830/003 ,  C12N2840/203 ,  C12N2840/445

公开(公告)号：EP1555874A2

公开(公告)日：2005-07-27

申请号：EP03774728.4

申请日：2003-10-09

申请人： Oxford Biomedica (UK) Limited

发明人： JOLLY, Doug ,  ROHLL, Jonathan ,  BUEHLER, Bernd ,  MITROPHANOUS, Kyri ,  RADCLIFF, Pippa

IPC分类号： A01N43/04 ,  A01N63/00 ,  A01N65/00 ,  A61K48/00 ,  C12N15/00 ,  C12N15/70 ,  C12N15/74

CPC分类号： C12N15/67 ,  C12N15/63 ,  C12N15/86 ,  C12N2740/15043 ,  C12N2830/003 ,  C12N2840/203 ,  C12N2840/445

摘要： Provided is an improved method for controlling gene expression in vivo through the use of a gene switch comprising one or more aptamer sequences operably linked to or incorporated into the untranslated regions (UTRs) of a transgene or nucleotide sequence of interest. Also provided are expression vectors having aptamer sequences located within the 3' UTR, the 5' UTR and between the genes of a multicistronic mRNA, as well as methods of using the expression vectors for controlling or regulating gene expression.

公开(公告)号：EP1443970A4

公开(公告)日：2005-01-12

申请号：EP01996590

申请日：2001-11-15

申请人： VASCULAR BIOGENICS LTD

发明人： HARATS DROR

IPC分类号： C12N15/09 ,  A61K31/7088 ,  A61K45/06 ,  A61K48/00 ,  A61P9/00 ,  A61P17/00 ,  A61P35/00 ,  A61P43/00 ,  C07K14/475 ,  C07K14/515 ,  C07K14/575 ,  C12N5/10 ,  C12N15/85

CPC分类号： C07K14/475 ,  A61K48/00 ,  A61K48/0058 ,  C07K14/515 ,  C07K14/57536 ,  C12N15/85 ,  C12N2830/001 ,  C12N2830/002 ,  C12N2830/008 ,  C12N2830/15 ,  C12N2830/42 ,  C12N2830/85 ,  C12N2840/445

摘要： An isolated polynucleotide functional as a promoter in eukaryotic cells is disclosed. The isolated polynucleotide includes an endothelial specific enhancer element as detailed herein. Further disclosed is a method of expressing a nucleic acid sequece of interest in endothelial cells.

公开(公告)号：EP1358203A2

公开(公告)日：2003-11-05

申请号：EP02714709.9

申请日：2002-01-08

申请人： Intronn Inc.

发明人： MITCHELL, Lloyd, G. ,  GARCIA-BLANCO, Mariano, A. ,  BAKER, Carl, C. ,  PUTTARAJU, Madaiah ,  MANSFIELD, Gary, S. ,  CHAO, Hengjun

IPC分类号： C07K1/00

CPC分类号： C12N15/85 ,  C12N15/1027 ,  C12N2800/108 ,  C12N2830/001 ,  C12N2830/60 ,  C12N2840/44 ,  C12N2840/445

摘要： The molecules and methods of the present invention provide a means for in vivo production of a trans-spliced molecule in a selected subset of cells. The pre-trans-splicing molecules of the invention are substrates for a trans-splicing reaction between the pre-trans-splicing molecules and a pre-mRNA which is uniquely expressed in the specific target cells. The in vivo trans-splicing reaction provides a novel mRNA which is functional as mRNA or encodes a protein to be expressed in the target cells. The expression product of the mRNA is a protein of therapeutic value to the cell or host organism a toxin which causes killing of the specific cells or a novel protein not normally present in such cells. The invention further provides PTMs that have been genetically engineered for the identification of exon/intron boundaries of pre-mRNA molecules using an exon tagging method. The PTMs of the invention can also be designed to result in the production of chimeric RNA encoding for peptide affinity purification tags which can be used to purify and identify proteins expressed in a specific cell type.

公开(公告)号：EP0883344A4

公开(公告)日：2002-08-07

申请号：EP96944760

申请日：1996-12-13

申请人： INTRONN LLC

发明人： MITCHELL LLOYD G

IPC分类号： C12N15/09 ,  A01N43/04 ,  A61K31/70 ,  A61K31/7105 ,  A61K31/711 ,  A61K38/00 ,  A61K48/00 ,  A61P31/12 ,  A61P35/00 ,  A61P37/06 ,  A61P43/00 ,  C07H21/02 ,  C07H21/04 ,  C07K14/34 ,  C12N5/10 ,  C12N15/00 ,  C12N15/10 ,  C12N15/11 ,  C12N15/63 ,  C12N15/70 ,  C12N15/74 ,  C12N15/85

CPC分类号： A61K48/005 ,  A61K38/00 ,  A61K48/00 ,  C07K14/34 ,  C12N15/1027 ,  C12N15/111 ,  C12N15/63 ,  C12N15/85 ,  C12N2320/33 ,  C12N2840/44 ,  C12N2840/445

公开(公告)号：EP1224313A1

公开(公告)日：2002-07-24

申请号：EP00970689.6

申请日：2000-10-06

申请人： University of Iowa Research Foundation

发明人： ENGELHARDT, John, F. ,  DUAN, Dongsheng ,  YAN, Ziyang

IPC分类号： C12N15/864 ,  C12N7/01 ,  C12N5/10 ,  A61K48/00

CPC分类号： C12N15/86 ,  A61K48/00 ,  C12N2750/14143 ,  C12N2800/108 ,  C12N2830/38 ,  C12N2840/203 ,  C12N2840/44 ,  C12N2840/445

摘要： The invention provides an isolated and purified DNA molecule comprising at least one DNA segment, a biologically active subunit or variant thereof, of a circular intermediate of adeno-associated virus, which DNA segment confers increased episomal stability, persistence or abundance of the isolated DNA molecule in a host cell. The invention also provides a composition comprising at least two adeno-associated virus vectors.