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1. 发明专利

JP2004065256A New transdominant tat variant of human immunodeficiency virus 审中-公开
公开(公告)号：JP2004065256A
公开(公告)日：2004-03-04
申请号：JP2003207954
申请日：2003-08-19
申请人： Transgene Sa , トランスジーンソシエテアノニム
发明人： MEHTALI MAJID , TANIA SORG
IPC分类号： C12N15/09 , A61K35/74 , A61K36/06 , A61K38/00 , A61K48/00 , A61P31/12 , A61P31/18 , C07K14/00 , C07K14/155 , C07K14/16 , C12N1/15 , C12N1/19 , C12N1/21 , C12N5/10 , C12N15/48 , C12N15/49 , C12P21/02 , C12R1/91 , A61K35/72
CPC分类号： C07K14/005 , A61K38/00 , A61K48/00 , A61K2039/525 , C12N2740/16322
摘要： PROBLEM TO BE SOLVED: To provide a transdominant variant of a HIV virus TAT protein containing at least one variation or a functional fragment of the protein. SOLUTION: The transdomonant variant comprises an HIV virus TAT protein or a functional fragment of the protein containing at least one mutation, wherein the mutation is an amino acid residue different from natural residue at positions 38, 40, 41, 45 or 47, and is different from alanine and threonine at the position 41 and different from alanine and histidine at the position 47. The method for producing the variant is provided, and a medicinal composition for treatment and prevention of HIV infection, using the variant as an effective ingredient, is provided. COPYRIGHT: (C)2004,JPO

2. 发明专利

JP2006320331A NOVEL IMPLANT AND NOVEL VECTOR FOR TREATMENT OF ACQUIRED DISEASE 审中-公开
公开(公告)号：JP2006320331A
公开(公告)日：2006-11-30
申请号：JP2006189518
申请日：2006-07-10
申请人： TRANSGENE SA
发明人： LEROY PIERRE , MEHTALI MAJID
IPC分类号： C12N15/09 , A61K9/00 , A61K35/76 , A61K38/00 , A61K39/395 , A61K48/00 , A61P31/04 , A61P31/18 , A61P35/00 , A61P37/04 , A61P43/00 , C07K16/10 , C07K16/30 , C12N5/10 , C12N7/00 , C12N15/861 , C12P21/08
摘要： PROBLEM TO BE SOLVED: To provide an adenoviral vector for treating and preventing cancer or AIDS. SOLUTION: The recombinant adenoviral vector comprises an exogenous nucleotide sequence encoding all or part of an antibody, and is placed under the control of various kinds of the elements necessary for its expression. The antibody is modified with a toxicity or immune enhancement agent. COPYRIGHT: (C)2007,JPO&INPIT

3. 发明专利

JP2009050267A Defective adenovirus and corresponding complimentation line 有权
标题翻译：有缺陷的ADENOVIRUS和相应的压迫线
公开(公告)号：JP2009050267A
公开(公告)日：2009-03-12
申请号：JP2008248884
申请日：2008-09-26
申请人： Transgene Sa , トランジェーヌ、ソシエテ、アノニムTransgene S.A.
发明人： IMLER JEAN-LUC , MEHTALI MAJID , PAVIRANI ANDREA
IPC分类号： C12N15/09 , A61K35/12 , A61K35/76 , A61K38/00 , A61K48/00 , C07K14/075 , C07K14/395 , C07K14/47 , C07K14/57 , C12N5/10 , C12N7/01 , C12N7/04 , C12N15/12 , C12N15/31 , C12N15/34 , C12N15/861
CPC分类号： C07K14/005 , A61K35/12 , A61K38/00 , A61K48/00 , C07K14/395 , C07K14/4712 , C07K14/57 , C12N7/00 , C12N15/86 , C12N2710/10322 , C12N2710/10343 , C12N2710/10352 , C12N2710/10362 , C12N2830/00 , C12N2830/002 , C12N2830/15 , C12N2830/60 , C12N2840/20
摘要： PROBLEM TO BE SOLVED: To provide a new defective adenovirus for the transfer and expression of an exogenous nucleotide sequence in a host cell or organism. SOLUTION: An adenovirus vector is provided, which is defective in replication, can be encapsidated in a complementation cell, and is derived by deletion, from an adenovirus genome containing, from 5' to 3', 5'ITR, encapsidated region, E1A region, E1B region, E2 region, E3 region, E4 region and 3'ITR, of (i) part or the whole of the E1A region and the whole of the E1B region encoding initial protein or (ii) part or the whole of the E1A region and part or the whole of at least one region selected from the E2 and E4 regions or (iii) part or the whole of the E1A region and part of the encapsidated region. COPYRIGHT: (C)2009,JPO&INPIT
摘要翻译：要解决的问题：提供用于在宿主细胞或生物体中转移和表达外源核苷酸序列的新的有缺陷的腺病毒。解决方案：提供了一种腺病毒载体，其复制缺陷，可以包被在互补细胞中，并且通过缺失从包含5'至3'，5'ITR，包被区的腺病毒基因组中衍生而来，E1A区域，E1B区域，E2区域，E3区域，E4区域和3'ITR，（i）部分或全部E1A区域和整个编码初始蛋白质的E1B区域，或（ii）部分或全部的E1A区域和选自E2和E4区域的部分或全部至少一个区域，或（iii）部分或全部E1A区域和部分封装区域。版权所有（C）2009，JPO＆INPIT

4. 发明专利

JP2008017849A Viral vectors and line for gene therapy 审中-公开
标题翻译：病毒载体和基因治疗线
公开(公告)号：JP2008017849A
公开(公告)日：2008-01-31
申请号：JP2007216267
申请日：2007-08-22
申请人： Transgene Sa , トランジェーヌ、ソシエテ、アノニムTransgene S.A.
发明人： MEHTALI MAJID , LUSKY MONIKA , RITTNER KAROLA
IPC分类号： C12N5/10 , C12N15/09 , A61K35/76 , A61K38/00 , A61K48/00 , A61P3/10 , A61P7/04 , A61P21/00 , A61P31/12 , A61P31/18 , A61P35/00 , A61P43/00 , C12N7/00 , C12N7/01 , C12N15/861
CPC分类号： C12N15/86 , A61K48/00 , C12N7/00 , C12N2710/10343 , C12N2710/10352 , C12N2830/006 , C12N2840/20
摘要： PROBLEM TO BE SOLVED: To provide novel viral vectors in which the expression of viral genes is regulated in such a way that it is functional in a complementation cell and non-functional in a host cell, as well as viral particles and host cells containing the novel vectors, and also a complementation cell including a viral gene expression repressor and a method for preparing infectious viral particles. SOLUTION: A complementation cell includes a DNA fragment coding an inducer and/or a repressor, and complements an adenoviral vector which is defective for the E1 function and at least one second, late or early adenoviral function. The cell comprises (i) a first cassette for the expression of all or part of the E1 region of an adenovirus, placed under the control of the elements necessary for its expression in the complementation cell, and (ii) a second cassette for the expression of all or part of a late or early region of an adenovirus other than the E1 region, placed under the control of the elements necessary for its expression in the complementation cell. COPYRIGHT: (C)2008,JPO&INPIT
摘要翻译：要解决的问题：提供新的病毒载体，其中病毒基因的表达以这样的方式被调节，使得其在互补细胞中是功能的，并且在宿主细胞中是非功能的，以及病毒颗粒和宿主含有新载体的细胞，以及包含病毒基因表达阻遏物的互补细胞以及制备感染性病毒颗粒的方法。补体细胞包括编码诱导物和/或阻抑物的DNA片段，并补充E1功能有缺陷的腺病毒载体和至少一个第二，晚期或早期腺病毒功能。所述细胞包含（i）用于表达腺病毒的全部或部分E1区域的第一盒，其置于其在互补细胞中表达所必需的元件的控制下，和（ii）用于表达的第二盒的全部或部分晚期或早期区域的E1区域以外的腺病毒，置于其在互补细胞中表达所必需的元件的控制下。版权所有（C）2008，JPO＆INPIT

5. 发明专利

JP2000106875A USE OF ADENOVIRUS E4 LEADING FRME FOR IMPROVING EXPRESSION OF TARGET GENE 失效
公开(公告)号：JP2000106875A
公开(公告)日：2000-04-18
申请号：JP22788099
申请日：1999-07-07
申请人： TRANSGENE SA
发明人： MEHTALI MAJID , LUSKY MONIKA
IPC分类号： C12N15/09 , A61K48/00 , A61P3/10 , A61P7/00 , A61P9/00 , A61P31/12 , A61P31/18 , A61P35/00 , A61P37/06 , C07K14/075 , C12N1/15 , C12N1/19 , C12N1/21 , C12N5/10 , C12N7/00 , C12N7/01 , C12N7/02 , C12N15/34 , C12N15/861
摘要： PROBLEM TO BE SOLVED: To obtain a new vector linked to regulatory element derived from an adenovirus genome in which E1 region is deleted or is non-functional and a part of the E4 region has been deleted and comprising a gene of interest linked to regulatory elements and retaining sufficient E4 sequences to improve persistence of expression of the gene. SOLUTION: This recombinant adenoviral vector is derived from an adenovirus genome in which at least all or part of the E1 region is deleted or is non- functional and a part of the E4 region has been deleted and comprise a gene of interest operably linked to regulatory elements, wherein the adenoviral vector retains sufficient E4 sequences to improve expression and/or persistence of expression of said gene of interest in a host cell or organism. The adenoviral vector can obtain long-term expression of the introduced gene in the cell and is useful for applications in liver specific gene therapy protocols because of low toxicity. The vector is obtained by using a polynucleotide comprising one or more open reading frames of the E4 region of an adenovirus.

6. 发明申请

WO0202765A2 CHIMERIC PROMOTERS FOR CONTROLLING EXPRESSION IN SMOOTH MUSCLE CELLS 审中-公开
标题翻译：用于控制在平滑肌细胞中表达的嵌合启动子
公开(公告)号：WO0202765A2
公开(公告)日：2002-01-10
申请号：PCT/EP0107657
申请日：2001-07-04
申请人： TRANSGENE SA , RIBAULT SEBASTIEN , NEUVILLE PASCAL , MEHTALI MAJID
发明人： RIBAULT SEBASTIEN , NEUVILLE PASCAL , MEHTALI MAJID
IPC分类号： A01K67/027 , A01K67/00 , A61K31/7088 , A61K35/76 , A61K38/00 , A61K38/21 , A61K38/43 , A61K48/00 , A61P9/00 , C07K14/47 , C12N5/10 , C12N7/00 , C12N15/09 , C12N15/11 , C12N15/85 , C12N15/86 , C12N15/861 , C12N15/00
CPC分类号： C12N15/85 , A61K48/00 , A61K48/0058 , C07K14/4716 , C12N9/1223 , C12N15/86 , C12N2710/10343 , C12N2799/022 , C12N2830/008 , C12N2830/15 , C12N2830/85
摘要： The present invention concerns a chimeric construct comprising a SMC-specific promoter operably linked to a muscle-specific enhancer. It also provides an expression cassette comprising such a chimeric construct to control expression of a therapeutic gene. Finally, the invention relates to a recombinant vector, a viral particle, an eukaryotic host cell, a composition comprising said expression cassette and their use for specific expression in SMCs and for therapeutic or prophylactic purposes, a method for the treatment of a human or animal organism as well as a transgenic non-human animal comprising integrated into its genome the chimeric construct, the expression cassette or the vector of the present invention.
摘要翻译：本发明涉及包含可操作地连接肌肉特异性增强子的SMC特异性启动子的嵌合构建体。它还提供了包含这种嵌合构建体的表达盒以控制治疗性基因的表达。最后，本发明涉及重组载体，病毒颗粒，真核宿主细胞，包含所述表达盒的组合物及其在SMC中特异性表达和用于治疗或预防目的的用途，治疗人或动物的方法包括在其基因组中整合有本发明的嵌合构建体，表达盒或载体的转基因非人动物。

7. 发明申请

WO9855639A3 RECOMBINANT ADENOVIRAL VECTORS COMPRISING A SPLICING SEQUENCE 审中-公开
标题翻译：包含突变序列的重组腺病毒载体
公开(公告)号：WO9855639A3
公开(公告)日：1999-03-04
申请号：PCT/FR9801105
申请日：1998-06-02
申请人： TRANSGENE SA , MEHTALI MAJID , LEROY PIERRE , MICHOU ANNE ISABELLE
发明人： MEHTALI MAJID , LEROY PIERRE , MICHOU ANNE-ISABELLE
IPC分类号： C12N15/09 , A61K48/00 , A61P43/00 , C07K14/55 , C07K14/745 , C12N5/10 , C12N15/12 , C12N15/26 , C12N15/86 , C12N15/861
CPC分类号： C12N15/86 , A61K48/00 , C12N2710/10343 , C12N2830/42 , C12N2840/44
摘要： The invention concerns a recombinant adenoviral vector driving from an adenovirus genome at least by deleting all or part of the E1 region, said adenoviral vector comprising an expression cassette of a gene of interest placed under the control of elements necessary for its expression in a host cell or a host organism, said elements required for its expression including at least a splicing sequence. The invention is characterised in that said splicing sequence is derived from a eukaryotic nuclear gene selected among the ovalbumen genes, α or β-globine, collagen and factor VIII of mammals or a synthetic splicing sequence. The invention also concerns a host cell and an infectious viral particle comprising such a vector, a method for preparing such a particle and their therapeutic or prophylactic use. The invention further concerns a pharmaceutical composition containing said adenoviral vector, said host cell or said viral particle.

8. 发明申请

WO0012741A9 INDUCIBLE EXPRESSION SYSTEM 审中-公开
标题翻译：可诱导表达系统
公开(公告)号：WO0012741A9
公开(公告)日：2001-03-29
申请号：PCT/FR9902051
申请日：1999-08-27
申请人： TRANSGENE SA , MEHTALI MAJID , SORG GUSS TANIA
发明人： MEHTALI MAJID , SORG-GUSS TANIA
IPC分类号： C12N15/09 , A61K35/76 , A61K38/00 , A61K48/00 , A61P3/10 , A61P7/04 , A61P9/00 , A61P9/08 , A61P31/00 , A61P31/04 , A61P31/12 , A61P31/18 , A61P35/00 , A61P43/00 , C12N1/15 , C12N1/19 , C12N5/10 , C12N7/00 , C12N15/861 , C12N15/62
CPC分类号： C12N15/86 , A61K48/00 , C07K2319/00 , C12N2710/10343 , C12N2830/00 , C12N2830/15
摘要： The invention concerns an inducible expression system using nucleotide sequences coding for a transcriptional activator of eukaryotic or viral origin and a recombinant adenoviral vector comprising a gene of interest placed under the control of a promoter inducible in trans by said transcriptional activator. The invention also concerns a recombinant adenoviral vector bearing a first expression cassette coding for a transcriptional activator and a second cassette bearing a gene of interest placed under the control of a promoter inducible in trans by said transcriptional activator. The invention further concerns an infectious viral particle, its preparation method, a eukaryotic cell and a pharmaceutical composition comprising such a vector or expression system as well as their use for therapeutic or prophylactic purposes.
摘要翻译：本发明涉及使用编码真核生物或病毒来源的转录激活子的核苷酸序列的诱导型表达系统，以及包含感兴趣的基因的重组腺病毒载体，其置于由所述转录激活子反转诱导的启动子控制下。本发明还涉及带有编码转录激活因子的第一表达盒的重组腺病毒载体和携带感兴趣的基因的第二个盒，所述第二个盒带有由所述转录激活子反向诱导的启动子控制。本发明还涉及感染性病毒颗粒，其制备方法，真核细胞和包含这种载体或表达系统的药物组合物及其用于治疗或预防目的的用途。

9. 发明申请

WO0012741A2 INDUCIBLE EXPRESSION SYSTEM 审中-公开
标题翻译：可诱导表达系统
公开(公告)号：WO0012741A2
公开(公告)日：2000-03-09
申请号：PCT/FR9902051
申请日：1999-08-27
申请人： TRANSGENE SA , MEHTALI MAJID , SORG GUSS TANIA
发明人： MEHTALI MAJID , SORG-GUSS TANIA
IPC分类号： C12N15/09 , A61K35/76 , A61K38/00 , A61K48/00 , A61P3/10 , A61P7/04 , A61P9/00 , A61P9/08 , A61P31/00 , A61P31/04 , A61P31/12 , A61P31/18 , A61P35/00 , A61P43/00 , C12N1/15 , C12N1/19 , C12N5/10 , C12N7/00 , C12N15/861 , C12N15/62
CPC分类号： C12N15/86 , A61K48/00 , C07K2319/00 , C12N2710/10343 , C12N2830/00 , C12N2830/15
摘要： The invention concerns an inducible expression system using nucleotide sequences coding for a transcriptional activator of eukaryotic or viral origin and a recombinant adenoviral vector comprising a gene of interest placed under the control of a promoter inducible in trans by said transcriptional activator. The invention also concerns a recombinant adenoviral vector bearing a first expression cassette coding for a transcriptional activator and a second cassette bearing a gene of interest placed under the control of a promoter inducible in trans by said transcriptional activator. The invention further concerns an infectious viral particle, its preparation method, a eukaryotic cell and a pharmaceutical composition comprising such a vector or expression system as well as their use for therapeutic or prophylactic purposes.
摘要翻译：本发明涉及使用编码真核生物或病毒来源的转录激活子的核苷酸序列的诱导型表达系统和包含感兴趣的基因的重组腺病毒载体，所述重组腺病毒载体置于由所述转录激活子反转诱导的启动子的控制之下。本发明还涉及携带编码转录激活因子的第一表达盒的重组腺病毒载体和携带感兴趣的基因的第二个盒，该基因置于由所述转录激活子反式诱导的启动子控制之下。本发明还涉及感染性病毒颗粒，其制备方法，真核细胞和包含这种载体或表达系统的药物组合物及其用于治疗或预防目的的用途。

10. 发明申请

WO9705255A3 HELPER VIRUSES FOR PREPARING RECOMBINANT VIRAL VECTORS 审中-公开
标题翻译：用于制备重组病毒载体的帮助病毒
公开(公告)号：WO9705255A3
公开(公告)日：1997-03-06
申请号：PCT/FR9601200
申请日：1996-07-30
申请人： TRANSGENE SA , LUSKY MONIKA , MEHTALI MAJID
发明人： LUSKY MONIKA , MEHTALI MAJID
IPC分类号： C12N15/09 , A61K35/76 , A61K48/00 , C12N5/10 , C12N7/00 , C12N7/01 , C12N15/56 , C12N15/861 , C12P21/00 , C12R1/91
CPC分类号： C12N7/00 , C12N15/86 , C12N2710/10343 , C12N2710/10352 , C12N2800/30
摘要： Novel helper vectors are provided for complementing defective recombinant viral vectors, characterised in that they are provided with recombination sequences recognised by a recombinase. A complementation cell expressing the recombinase, and a method for preparing recombinant viral vectors as infectious viral particles for transferring and expressing genes of interest in a host organism or cell, are also provided. The invention is particularly suitable for use in gene therapy, especially in humans.

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