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1. 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。

2. 发明申请

WO2006056617A1 GENE TRAP CASSETTES FOR RANDOM AND TARGETED CONDITIONAL GENE INACTIVATION 审中-公开
标题翻译：用于随机和有针对性的条件基因失活的基因捕获系统
公开(公告)号：WO2006056617A1
公开(公告)日：2006-06-01
申请号：PCT/EP2005/056282
申请日：2005-11-28
申请人： FRANKGEN BIOTECHNOLOGIE AG , GSF FORSCHUNGSZENTRUM FÜR UMWELT UND GESUNDHEIT GMBH , MPG MAX-PLANCK-GESELLSCHAFT ZUR FÖRDERUNG DER WISSENSCHAFTEN E.V. , VON MELCHNER, Harald , SCHNÜTGEN, Frank , WURST, Wolfgang , RUIZ, Patricia , DE-ZOLT, Silke , FLOSS, Thomas , HANSEN, Jens
发明人： VON MELCHNER, Harald , SCHNÜTGEN, Frank , WURST, Wolfgang , RUIZ, Patricia , DE-ZOLT, Silke , FLOSS, Thomas , HANSEN, Jens
IPC分类号： C12N15/90 , A01K67/027 , C12N5/10
CPC分类号： C12N15/1051 , C12N15/907 , C12N2799/027 , C12N2800/30 , C12N2800/60
摘要： The present invention provides for a new type of gene trap cassettes, which can induce conditional mutations. The cassettes rely on directional site-specific recombination systems, which can repair and re-induce gene trap mutations when activated in succession. After the gene trap cassettes are inserted into the genome of the target organism, mutations can be activated at a particular time and place in somatic cells. In addition to their conditional features, the gene trap cassettes create multipurpose alleles amenable to a wide range of post-insertional modifications. Such gene trap cassettes can be used to mutationally inactivate all cellular genes. In addition the invention relates to a cell, preferably a mammalian cell which contains the above mentioned gene trap cassette. Moreover, the invention relates to the use of said cell for identification and/or isolation of genes and for the creation of transgenic organisms to study gene function at various developmental stages, including the adult, as well as for the creation of animal models of human disease useful for in vivo drug target validation. In conclusion, the present invention provides a process which enables a temporally and/or spatially restricted inactivation of all genes that constitute a living organism.
摘要翻译：本发明提供了可诱导条件突变的新型基因捕获盒。盒子依赖于定向位点特异性重组系统，其可以在连续激活时修复和再诱导基因陷阱突变。将基因陷阱盒插入目标生物体的基因组后，可以在特定时间激活突变并置于体细胞中。除了它们的条件特征之外，基因陷阱盒创建了多用途等位基因，适用于大量的插入后修饰。这种基因捕获盒可用于突变灭活所有细胞基因。此外，本发明涉及含有上述基因捕获盒的细胞，优选哺乳动物细胞。此外，本发明涉及所述细胞用于鉴定和/或分离基因和用于产生转基因生物以研究包括成年人在内的各种发育阶段的基因功能，以及用于创建人的动物模型用于体内药物靶标验证的疾病。总之，本发明提供一种使构成活生物体的所有基因的时间和/或空间限制性失活的方法。

3. 发明申请

WO2006056615A1 ENHANCER-CONTAINING GENE TRAP VECTORS FOR RANDOM AND TARGETED GENE TRAPPING 审中-公开
标题翻译：用于随机和有针对性的基因捕获的含增强基因捕获载体
公开(公告)号：WO2006056615A1
公开(公告)日：2006-06-01
申请号：PCT/EP2005/056278
申请日：2005-11-28
申请人： FRANKGEN BIOTECHNOLOGIE AG , GSF FORSCHUNGSZENTRUM FÜR UMWELT UND GESUNDHEIT GMBH , MPG MAX-PLANCK-GESELLSCHAFT ZUR FÖRDERUNG DER WISSENSCHAFTEN E.V. , VON MELCHNER, Harald , SCHNÜTGEN, Frank , WURST, Wolfgang , RUIZ, Patricia
发明人： VON MELCHNER, Harald , SCHNÜTGEN, Frank , WURST, Wolfgang , RUIZ, Patricia
IPC分类号： C12N15/867 , C12N15/85 , C12N15/10
CPC分类号： C12N15/1051 , C12N15/63 , C12N2740/13043 , C12N2800/30 , C12N2800/60
摘要： The present invention relates to a novel class of gene trap vector (enhanced gene trap vectors, eGTV) for efficiently identifying silent or weakly expressed target genes in mammalian genomes, methods of their production and methods for identifying and mutating target genes by using the enhanced gene trap vectors. The gene trap vectors of the present invention can also be used for inducing the expression of silent genes and enhancing the expression of weakly expressed genes. The use of the enhanced gene trap vectors for creating transgenic organisms to identify gene function and to validate pharmaceutical compounds prior to clinical applications is a further aspect of the present invention.
摘要翻译：本发明涉及用于有效识别哺乳动物基因组中的沉默或弱表达的靶基因的新型基因捕获载体（增强型基因捕获载体，eGTV），其生产方法以及通过使用增强型基因鉴定和突变靶基因的方法陷阱向量。本发明的基因捕获载体也可用于诱导沉默基因的表达和增强弱表达基因的表达。使用增强型基因捕获载体产生转基因生物以鉴定基因功能并在临床应用之前验证药物化合物是本发明的另一方面。

4. 发明申请

WO1998024918A1 GENE TRAP CONSTRUCT FOR IDENTIFICATION AND ISOLATION OF GENES 审中-公开
标题翻译：基因捕获构建鉴定及基因的分离
公开(公告)号：WO1998024918A1
公开(公告)日：1998-06-11
申请号：PCT/EP1997006816
申请日：1997-12-05
申请人： HÖLZER, Dieter , VON MELCHNER, Harald
发明人： HÖLZER, Dieter
IPC分类号： C12N15/65
CPC分类号： C12Q1/6897 , C12N15/1051
摘要： The relation relates to a gene trap construct containing a first reporter gene, which, after activation, can activate a second reporter gene, and the use of this gene trap construct for identification and/or isolation of genes, in particular transiently expressed genes. The invention relates further to a cell, preferably a mammal cell, containing the above-mentioned gene trap construct. The invention relates furthermore to the use of this mammal cell for identification and/or isolation of genes, in particular transient genes. In addition, the invention relates to a vector containing the above mentioned gene trap construction, as well as a kit for identifying and/or isolating genes, in particular transient genes, which contains at least the above-mentioned gene trap construct or the above-mentioned vector. Finally, the invention relates to a method for the identification and/or isolation of genes, in particular transient genes.
摘要翻译：本发明涉及一种基因捕获构建体，包含第一报告基因，它可以激活在激活时和使用该基因构建体捕获用于识别和/或基因的分离的第二报道基因，尤其是瞬时表达的基因。本发明还涉及一种细胞，优选包含上述基因捕获构建体的哺乳动物细胞。本发明还涉及使用用于识别和/或基因的隔离这些哺乳动物细胞，特别是瞬态的基因。此外，本发明涉及含有上述基因捕获构建体，以及用于鉴定和/或基因的分离的试剂盒，特别是瞬态源基因的载体，至少含有上述基因捕获构建体或载体上面提到。最后，本发明涉及用于鉴定和/或基因的分离，特别是瞬态的基因的方法。

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