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1. 发明申请

WO2007008309A3 STEM AND PROGENITOR CELL EXPANSION BY EVI, EVI-LIKE GENES AND SETBP1 审中-公开
标题翻译：由EVI，EVI样基因和SETBP1进行干细胞和前列腺细胞扩增
公开(公告)号：WO2007008309A3
公开(公告)日：2007-07-26
申请号：PCT/US2006021413
申请日：2006-06-01
申请人： CHILDRENS HOSP MEDICAL CENTER , VON KALLE CHRISTOF , SCHMIDT MANFRED , GREZ MANUEL
发明人： VON KALLE CHRISTOF , SCHMIDT MANFRED , GREZ MANUEL
IPC分类号： C12N15/10 , C12N5/10 , C12N15/861
CPC分类号： C12N5/0634 , A61K48/00 , C12N2501/998 , C12N2510/00 , C12N2799/027
摘要： A method of increasing cell proliferation by modulating levels of EVI and related genes. Activation of EVI-1, PRDM16, or SETBP1 can increase the proliferation rate, self renewal and/or in vitro and/or in vivo survival and/or engraftment of human cells, either in vitro or in vivo. The gene modulation can be performed by various means, including traditional cloning methods and retroviral-based gene activation methods. The method can also be used to more efficiently deliver gene-corrected cells to a patient in need of treatment.
摘要翻译：通过调节EVI和相关基因水平来增加细胞增殖的方法。 EVI-1，PRDM16或SETBP1的激活可以在体外或体内增加人细胞的增殖速率，自我更新和/或体外和/或体内存活和/或植入。基因调节可以通过各种方式进行，包括传统的克隆方法和基于逆转录病毒的基因活化方法。该方法还可以用于更有效地将基因校正的细胞递送至需要治疗的患者。

2. 发明申请

WO1997007223A1 SELF-DELETING VECTORS FOR GENE THERAPY 审中-公开
标题翻译：用于基因治疗的自我删除载体
公开(公告)号：WO1997007223A1
公开(公告)日：1997-02-27
申请号：PCT/EP1996000761
申请日：1996-02-23
申请人： VON MELCHNER, Harald , RUSS, Andreas, Peter , GREZ, Manuel
IPC分类号： C12N15/86
CPC分类号： C12N15/86 , A61K48/00 , C12N2740/13043
摘要： The invention involves the development of vectors for somatic gene therapy. The vectors transduce a complete transcriptional unit containing a promoter, a protein coding sequence and a polyadenylation sequence into the genome of mammalian cells. Upon integration, the vectors delete most viral and non-viral sequences unrelated to transcriptional unit thus avoiding common problems encountered with conventional retrovirus vectors such as repression of gene expression by transcriptional silencing, mobilization of endogenous retroviruses, activation of oncogenes or development of an immune response. The invention exploits (i) the natural life cycle of retroviruses, involving duplication of the terminal control regions U5 and U3 to generate long terminal repeats (LTR) and (ii) the ability of site specific recombinases to excise any sequences positioned between two specific target sequences from the mammalian genome. Thus, the retroviruses of the invention transduce the coding sequences of a site specific recombinase and at least one recombinase-specific target sequence into the genome along with the transcriptional unit expressing a therapy gene.
摘要翻译：本发明涉及体细胞基因治疗载体的开发。载体将含有启动子，蛋白质编码序列和多聚腺苷酸化序列的完整转录单位转导到哺乳动物细胞的基因组中。在整合时，载体删除与转录单位无关的大多数病毒和非病毒序列，从而避免常规逆转录病毒载体遇到的常见问题，例如通过转录沉默抑制基因表达，动员内源性逆转录病毒，致癌基因的活化或发展免疫应答。本发明利用（i）逆转录病毒的自然生命周期，涉及终端控制区域U5和U3的复制以产生长末端重复序列（LTR）和（ii）位点特异性重组酶能够切除位于两个特异性靶标来自哺乳动物基因组的序列。因此，本发明的逆转录病毒与表达治疗基因的转录单位一起将位点特异性重组酶和至少一种重组酶特异性靶序列的编码序列转导入基因组。

3. 发明申请

WO1994005327A1 NEW HIV-1 ISOLATES OF A SUBTYPE AND ITS DIFFERENTIAL DIAGNOSIS, VACCINE AGAINST HIV-1 INFECTION OF THIS SUBTYPE AND PROCESS FOR PREPARING AND USING THE HIV-1 ISOLATE 审中-公开
标题翻译：新的HIV-1病毒株亚型和鉴别诊断，疫苗的抗HIV-1病毒感染这个亚型及其制备方法，采用了HIV-1病毒株
公开(公告)号：WO1994005327A1
公开(公告)日：1994-03-17
申请号：PCT/EP1993002275
申请日：1993-08-25
申请人： CHEMOTHERAPEUTISCHES FORSCHUNGSINSTITUT , DIETRICH, Ursula , VON BRIESEN, Hagen , GREZ, Manuel , RÜBSAMEN-WAIGMANN, Helga
发明人： CHEMOTHERAPEUTISCHES FORSCHUNGSINSTITUT
IPC分类号： A61K39/21
CPC分类号： C07K14/005 , A61K39/00 , C12N7/00 , C12N2740/15022 , C12N2740/16021 , C12N2740/16122
摘要： Three new HIV-1 isolates HIV-1D747 (ECACC V92082718), HIV-1D757 (ECACC V92082719) and HIV-1D760 (ECACC V92082720) are disclosed, which represent a further indenpendent subtype of the HIV-1 family and have been recovered from Indian patients which at the time when the virus was isolated showed no typical AIDS symptoms. Also disclosed are vaccines against HIV-1 infections by this subtype and a process for producing the same, as well as the use of the HIV-1 isolates to produce protective vaccines against HIV-1 infections and for therapeutical use after HIV-1 infection, as well as for differential diagnosis.

4. 发明申请

WO2009012959A1 GENE THERAPY OF CHRONIC GRANULOMATOUS DISEASE 审中-公开
标题翻译：慢性粒细胞病的基因治疗
公开(公告)号：WO2009012959A1
公开(公告)日：2009-01-29
申请号：PCT/EP2008/005956
申请日：2008-07-21
申请人： VISION 7 GMBH , CHEMOTHERAPEUTISCHES FORSCHUNGSINSTITUT GEORG-SPEYER-HAUS , STEIN, Stefan , GREZ, Manuel , SCHAMBACH, Axel
发明人： STEIN, Stefan , GREZ, Manuel , SCHAMBACH, Axel
IPC分类号： C12N9/02 , A61K48/00
CPC分类号： C12N15/86 , A61K48/00 , C12N9/0038 , C12N2740/13043 , C12N2800/22 , C12N2830/008
摘要： The present invention relates to the field of gene therapy. In particular, it provides a nucleic acid encoding gp91phox, wherein codon-usage of said gp91phox nucleic acid is optimized for expression in human cells, and an expression construct, in particular a retroviral vector suitable for gene therapy of chronic granulomatous disease, comprising said nucleic acid. Additionally, methods of preparing cells comprising the construct, said cells and pharmaceutical compositions comprising the construct or cells of the invention are provided.
摘要翻译：本发明涉及基因治疗领域。特别地，其提供编码gp91phox的核酸，其中所述gp91phox核酸的密码子使用被优化用于在人细胞中表达，以及表达构建体，特别是适用于慢性肉芽肿病的基因治疗的逆转录病毒载体，其包含所述核酸酸。此外，提供了制备包含构建体，所述细胞和包含本发明的构建体或细胞的药物组合物的细胞的方法。

5. 发明申请

WO2011135002A1 GENE TRAPPING FOR USE IN GENE THERAPY 审中-公开
标题翻译：在基因治疗中使用的基因诱捕
公开(公告)号：WO2011135002A1
公开(公告)日：2011-11-03
申请号：PCT/EP2011/056665
申请日：2011-04-27
申请人： MELCHNER, Harald von , GREZ, Manuel , SCHNÜTGEN, Frank , SCHULTZE-STRASSER, Stephan
发明人： MELCHNER, Harald von , GREZ, Manuel , SCHNÜTGEN, Frank , SCHULTZE-STRASSER, Stephan
IPC分类号： C12N15/10 , A61K48/00 , C12N9/02 , C12N15/867
CPC分类号： C12N15/86 , C07K2319/50 , C12N9/0036 , C12N2740/13043 , C12N2800/24 , C12N2800/60 , C12N2830/008 , C12N2840/20 , C12N2840/203
摘要： The present invention provides gene trap constructs for use in gene therapy, notably gene trap constructs for the correction of the Gp91phox (Nox2) mutation in chronic granulomatous disease (X-CGD).
摘要翻译：本发明提供用于基因治疗的基因陷阱构建体，特别是用于校正慢性肉芽肿病（X-CGD）中的Gp91phox（Nox2）突变的基因陷阱构建体。

6. 发明申请

WO2007008309A2 STEM AND PROGENITOR CELL EXPANSION BY EVI, EVI-LIKE GENES AND SETBP1 审中-公开
标题翻译： EVI，EVI-LIKE基因和SETBP1的干细胞和前体细胞扩增
公开(公告)号：WO2007008309A2
公开(公告)日：2007-01-18
申请号：PCT/US2006/021413
申请日：2006-06-01
申请人： CHILDREN'S HOSPITAL MEDICAL CENTER , VON KALLE, Christof , SCHMIDT, Manfred , GREZ, Manuel
发明人： VON KALLE, Christof , SCHMIDT, Manfred , GREZ, Manuel
IPC分类号： C12N15/10 , C12N5/10 , C12N15/861
CPC分类号： C12N5/0634 , A61K48/00 , C12N2501/998 , C12N2510/00 , C12N2799/027
摘要： A method of increasing cell proliferation by modulating levels of EVI and related genes. Activation of EVI-1, PRDM16, or SETBP1 can increase the proliferation rate, self renewal and/or in vitro and/or in vivo survival and/or engraftment of human cells, either in vitro or in vivo. The gene modulation can be performed by various means, including traditional cloning methods and retroviral-based gene activation methods. The method can also be used to more efficiently deliver gene-corrected cells to a patient in need of treatment.
摘要翻译：通过调节EVI和相关基因水平来增加细胞增殖的方法。 EVI-1，PRDM16或SETBP1的活化可以在体外或体内增加人细胞的增殖速率，自我更新和/或体外和/或体内存活和/或植入。基因调控可以通过各种手段进行，包括传统的克隆方法和基于逆转录病毒的基因激活方法。该方法还可用于更有效地将基因校正的细胞递送至需要治疗的患者。

7. 发明申请

WO2004092355A1 EXPRESSION OF PROTEINS IN CORD BLOOD-DERIVED ENDOTHELIAL CELLS 审中-公开
标题翻译：蛋白质在牙髓衍生的内皮细胞中的表达
公开(公告)号：WO2004092355A1
公开(公告)日：2004-10-28
申请号：PCT/EP2004/003998
申请日：2004-04-15
申请人： DRK-BLUTSPENDEDIENST BADEN-WÜ RTTEMBERGHESSEN GGMBH , HERDER, Christian , TONN, Torsten , GREZ, Manuel , SEIFRIED, Erhard
发明人： HERDER, Christian , TONN, Torsten , GREZ, Manuel , SEIFRIED, Erhard
IPC分类号： C12N5/06
CPC分类号： C12N5/069 , C07K14/755 , C12N2501/10 , C12N2501/115 , C12N2501/125 , C12N2501/165 , C12N2501/998 , C12N2510/02 , C12N2799/021 , C12N2799/027
摘要： The invention provides a population of mature endothelial cells derived from human cord blood. The cells can be transduced with DNA encoding a therapeutically effective protein such as a blood coagulation factor. The cells are useful in a method for the production of a blood coagulation factor. They can further be used in hemophilia A or B gene therapy.
摘要翻译：本发明提供了源自人脐带血的成熟内皮细胞群。可以用编码治疗有效的蛋白质如血液凝固因子的DNA转导细胞。细胞在生产凝血因子的方法中是有用的。它们可以进一步用于血友病A或B基因治疗。

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