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1. 发明申请

US20180265890A1 EFFICIENT AND SAFE TRANSPOSON INTEGRATION SYSTEM AND USE THEREOF 审中-公开
公开(公告)号：US20180265890A1
公开(公告)日：2018-09-20
申请号：US15764840
申请日：2016-09-14
申请人： SHANGHAI CELL THERAPY RESEARCH INSTITUTE , SHANGHAI ENGINEERING RESEARCH CENTER FOR CELL THERAPY GROUP CO., LTD
发明人： Qijun QIAN , Huajun JIN , Linfang LI , Tao LIU , Minghui ZUO , Hongping WU , Mengchao WU
IPC分类号： C12N15/85 , C12N5/16 , C12N15/52 , C12N15/113
CPC分类号： C12N15/85 , C12N5/16 , C12N15/113 , C12N15/52 , C12N2015/8518 , C12N2800/107
摘要： The invention belongs to the field of molecular biology, and relates to an efficient and safe transposon integration system and use thereof. The invention also relates to a nucleic acid construct and use thereof. Preferably, the nucleic acid construct comprises the following elements in order: a 5′-terminal repeat sequence of a transposon, a multiple cloning site, a polyA tailing signal sequence, a 3′-terminal repeat sequence of a transposon, a sequence encoding a transposase and a promoter controlling expression of the transposase; wherein the multiple cloning site is used for operably inserting an exogenous gene and optionally a promoter controlling expression of the exogenous gene; the polyA tailing signal sequence has a polyA tailing signal function in both forward and reverse directions; and the direction of the expression cassette of the transposase is opposite to the direction of the exogenous gene expression cassette. The nucleic acid construct is useful for mediating efficient and safe expression of an exogenous gene in a host cell.

2. 发明申请

US20180105833A1 SYSTEM FOR STABLE GENE EXPRESSION 审中-公开
公开(公告)号：US20180105833A1
公开(公告)日：2018-04-19
申请号：US15558899
申请日：2016-03-16
申请人： Andrew WILBER , Board of Trustees, Southern Illinois University
发明人： Andrew WILBER , William Halford
IPC分类号： C12N15/85 , C12N15/63
CPC分类号： C12N15/85 , C12N15/63 , C12N15/635 , C12N15/79 , C12N2800/107 , C12N2800/60 , C12N2800/70 , C12N2800/80 , C12N2800/90 , C12N2830/205
摘要： An inducible expression cassette for controlled expression of multiple genes includes the DNA sequences of a bidirectional promoter inserted between two DNA sequences. Transfection into a host cell and the two DNA sequences encode genes of interest, provides one DNA sequence expressed constitutively and manipulation of the expression of the other DNA sequence. A regulatory DNA cassette containing another bidirectional promoter and two DNA sequences that encode a marker and a regulatory expression product is also disclosed. Both cassettes can be incorporated in a non-viral vector, like the Sleeping Beauty transposon, or a viral vector to induce controlled expression of multiple genes into host cells. A kit containing a package of each above vector type is also disclosed, as is a method of transforming a host cell.

3. 发明授权

US09777276B2 ADAM12 inhibitors and their use against inflammation-induced fibrosis 有权
公开(公告)号：US09777276B2
公开(公告)日：2017-10-03
申请号：US14479006
申请日：2014-09-05
申请人： Institut Pasteur
发明人： Lucie Peduto , Gerard Eberl
IPC分类号： A61K39/395 , A61K39/00 , C12N15/113 , A61K38/48 , G01N33/573 , G01N33/68 , A01K67/027 , C12Q1/68 , C12N15/85
CPC分类号： C12N15/1137 , A01K67/0275 , A01K2217/203 , A01K2217/206 , A01K2227/105 , A01K2267/0368 , A01K2267/0393 , A61K38/4886 , C12N15/8509 , C12N2015/859 , C12N2310/14 , C12N2320/30 , C12N2800/107 , C12N2820/55 , C12Q1/6883 , C12Q1/6897 , C12Q2600/158 , C12Y304/24 , G01N33/573 , G01N33/6893 , G01N2333/96486 , G01N2500/00 , G01N2800/085 , G01N2800/12 , G01N2800/382
摘要： The present invention relates to the field of fibrosis and inflammation and more particularly to the use of ADAM12 (A Disintegrin and Metalloproteinase 12) inhibitors to prevent or treat inflammation-induced fibrosis. The present invention also relates to the use of ADAM12 as a marker for inflammation-induced fibrosis and to the ablation of ADAM12 expressing cells as therapeutic approach to interfere with the development of pro-fibrotic cells.

4. 发明申请

US20170240907A1 PRODUCTION OF RECOMBINANT EXPRESSION VECTORS 审中-公开
公开(公告)号：US20170240907A1
公开(公告)日：2017-08-24
申请号：US15590902
申请日：2017-05-09
申请人： Eberhard Karls Universitaet Tuebingen Medizinische Fakultaet
发明人： Ralf AMANN , Hans-Joachim RZIHA
IPC分类号： C12N15/64 , C12N15/10 , C07K14/005 , C12N15/90 , C07K14/73
CPC分类号： C12N15/64 , A61K39/0011 , A61K39/12 , C07K14/005 , C07K14/70514 , C12N15/1013 , C12N15/907 , C12N2760/20134 , C12N2800/107
摘要： The present invention relates to a method for the production of recombinant expression vectors, a kit adapted to carrying out the method, a vector used in the context of the method, a cell containing such vector and the use of the vector.

5. 发明授权

US09657310B2 Expression vector 有权
公开(公告)号：US09657310B2
公开(公告)日：2017-05-23
申请号：US14396929
申请日：2013-04-25
申请人： JCR Pharmaceuticals Co., Ltd.
发明人： Kenichi Takahashi , Shinji Kakimoto
IPC分类号： C12N15/00 , C12N9/00 , C12N15/85 , C12N15/52 , C12N9/06 , C07K14/59 , C07K14/505 , C07K16/28
CPC分类号： C12N15/85 , C07K14/505 , C07K14/59 , C07K16/2887 , C07K2317/14 , C12N9/003 , C12N9/93 , C12N15/52 , C12N2800/107 , C12N2830/00 , C12N2830/42 , C12N2830/50 , C12N2830/60 , C12N2840/60 , C12Y105/01003 , C12Y603/01002
摘要： Disclosed are a novel expression vector for efficient expression of recombinant proteins in mammalian cells, a mammalian cell transformed with the vector, and a method for production of the mammalian cell. The expression vector is an expression vector for expression of a mammalian protein and includes a gene expression regulatory site, and a gene encoding the protein downstream thereof, and an internal ribosome entry site further downstream thereof, and a gene encoding a glutamine synthetase further downstream thereof, and a dihydrofolate reductase gene downstream of either the same gene expression regulatory site or another gene expression regulatory site in addition to the former.

6. 发明申请

US20170114363A1 CHIMERIC POST-TRANSCRIPTIONAL REGULATORY ELEMENT 审中-公开
公开(公告)号：US20170114363A1
公开(公告)日：2017-04-27
申请号：US15335930
申请日：2016-10-27
申请人： Celltheon Corporation
发明人： Nikhil Goel , Amita Goel
IPC分类号： C12N15/85 , C07K16/28
CPC分类号： C12N15/85 , C07K16/2887 , C12N2800/107 , C12N2830/30 , C12N2830/48 , C12N2830/60
摘要： The present disclosure relates to chimeric post-transcriptional regulatory elements (PRE) and vectors useful for expressing a protein in a cell. The PRE contains alpha, beta and optionally gamma subelements selected from different native PRE sequences and are discovered to be more potent than their native counterparts.

7. 发明申请

US20170088844A1 DNA Plasmids with Improved Expression 审中-公开
公开(公告)号：US20170088844A1
公开(公告)日：2017-03-30
申请号：US15375215
申请日：2016-12-12
申请人： Nature Technology Corporation
发明人： James A. WILLIAMS
IPC分类号： C12N15/63 , C12N15/70
CPC分类号： C12N15/635 , A61K39/00 , A61K48/00 , C12N15/70 , C12N15/85 , C12N2800/107 , C12N2820/55
摘要： The present invention relates to the production and use of covalently closed circular (ccc) recombinant plasmids, and more particularly to vector modifications that improve expression of said DNA molecules in the target organism.

8. 发明申请

US20160304883A1 ARTIFICIAL NUCLEIC ACID MOLECULES 审中-公开
标题翻译：人工核酸分子
公开(公告)号：US20160304883A1
公开(公告)日：2016-10-20
申请号：US15195524
申请日：2016-06-28
申请人： CureVac AG
发明人： Stefanie GRUND , Thomas SCHLAKE
IPC分类号： C12N15/68 , C12N15/85
CPC分类号： C12N15/68 , A61K39/00 , A61K48/00 , C12N15/67 , C12N15/85 , C12N2800/107 , C12N2800/22 , C12N2830/00 , C12N2830/50 , C12N2840/203
摘要： The invention relates to an artificial nucleic acid molecule comprising at least one open reading frame and at least one 3′-untranslated region element (3′-UTR element) comprising a nucleic acid sequence which is derived from the 3′-UTR of a FIG4 gene or from a variant of the 3′-UTR of a FIG4 gene. The invention further relates to the use of such an artificial nucleic acid molecule in gene therapy and/or genetic vaccination. Furthermore, the invention relates to the use of a 3′-UTR element comprising a nucleic acid sequence which is derived from the 3′-UTR of a FIG4 gene or from a variant of the 3′-UTR of a FIG4 gene for the stabilization and/or prolongation of protein expression from a nucleic acid sequence comprising such 3′-UTR element.
摘要翻译：本发明涉及包含至少一个开放阅读框和至少一个3'非翻译区域元件（3'-UTR元件）的人造核酸分子，其包含衍生自图4的3'-UTR的核酸序列基因或来自FIG4基因的3'-UTR的变体。本发明还涉及这种人工核酸分子在基因治疗和/或基因疫苗接种中的用途。此外，本发明涉及包含衍生自图4基因的3'-UTR或FIG4基因的3'-UTR的变体的核酸序列用于稳定化的3'-UTR元件的用途和/或来自包含这种3'-UTR元件的核酸序列的蛋白质表达的延长。

9. 发明申请

US20160222416A1 Orthogonal Cas9 Proteins for RNA-Guided Gene Regulation and Editing 审中-公开
公开(公告)号：US20160222416A1
公开(公告)日：2016-08-04
申请号：US14903728
申请日：2014-07-08
申请人： PRESIDENT AND FELLOWS OF HARVARD COLLEGE
发明人： George M. Church , Kevin M. Esvelt , Prashant G. Mali
IPC分类号： C12N15/90 , C12N15/85 , C07K2/00 , C12N9/22 , C12N15/11
CPC分类号： C12N15/907 , C07K2/00 , C12N9/22 , C12N15/11 , C12N15/113 , C12N15/85 , C12N2310/3519 , C12N2320/33 , C12N2320/50 , C12N2800/107 , C12N2800/40 , C12N2800/80 , C12N2810/10 , C12N2830/00 , C12N2830/34 , C12N2999/007 , C12Y301/00
摘要： Methods of modulating expression of a target nucleic acid in a cell are provided including use of multiple orthogonal Cas9 proteins to simultaneously and independently regulate corresponding genes or simultaneously and independently edit corresponding genes.

10. 发明申请

US20160220699A1 GENE THERAPY FOR THE REGENERATION OF CHONDROCYTES OR CARTILAGE TYPE CELLS 审中-公开
标题翻译：用于再生念珠菌或细胞培养细胞的基因治疗
公开(公告)号：US20160220699A1
公开(公告)日：2016-08-04
申请号：US14917560
申请日：2014-09-09
申请人： FIGENE, LLC
发明人： Pete O'Heeron
IPC分类号： A61K48/00
CPC分类号： A61K48/0058 , A61K48/0075 , C12N15/85 , C12N2800/107
摘要： Embodiments of the invention concern the regeneration of chondrocytes and cartilage-type cells. In certain embodiments, one or more genes are employed for the regeneration of chondrocytes and cartilage-type cells. In particular embodiments, one or more gene therapy regimens are employed for the regeneration of chondrocytes and cartilage-type cells. In particular aspects, embodiments concern cartilage repair, such as articular cartilage repair. More particularly, embodiments for the disclosure concern using gene therapy for the attraction, generation and/or regeneration of chondrocytes or other cartilage-type cells and/or the generation and/or repair of cartilage tissue. In specific embodiments of the disclosure, gene therapy is provided that is capable of attracting and/or generating desired cells in vivo.
摘要翻译：本发明的实施方案涉及软骨细胞和软骨型细胞的再生。在某些实施方案中，一种或多种基因用于软骨细胞和软骨型细胞的再生。在具体实施方案中，一种或多种基因治疗方案用于软骨细胞和软骨型细胞的再生。在具体方面，实施例涉及软骨修复，例如关节软骨修复。更具体地，涉及使用基因治疗用于软骨细胞或其它软骨型细胞的吸引，产生和/或再生和/或软骨组织的产生和/或修复的公开内容的实施方案。在本公开的具体实施方案中，提供能够在体内吸引和/或产生所需细胞的基因治疗。

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