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1. 发明申请

WO03093414A2 TRANSGENIC ANIMAL MODEL FOR SPLICEOSOME-MEDIATED RNA TRANS-SPLICING 审中-公开
标题翻译：用于SPLICEOSOME介导RNA转移分离的转基因动物模型
公开(公告)号：WO03093414A2
公开(公告)日：2003-11-13
申请号：PCT/US0313161
申请日：2003-04-30
申请人： INTRONN INC , PUTTARAJU MADAIAH , MITCHEL LLOYD , ENGELHARDT JOHN F , BLANCO MARIANO GARCIA , LIU XIAOMING
发明人： PUTTARAJU MADAIAH , MITCHEL LLOYD , ENGELHARDT JOHN F , BLANCO MARIANO GARCIA , LIU XIAOMING
IPC分类号： A01K67/027 , C12N15/09 , C12N15/85 , C12Q1/68 , C12N
CPC分类号： C12N15/8509 , A01K67/0275 , A01K2217/05 , A01K2227/105 , A01K2267/0393 , C12N2800/30
摘要： The present invention relates to development of an animal model system for in vivo testing of spliceosome-mediated RNA trans-splicing reactions. The present invention provides transgenic animals, and methods for generating such animals, that have been genetically engineered to expresses a target precursor messenger RNA molecule (target pre-mRNA) that serves as a substrate for a trans-splicing reaction. Specifically, the transgenic animals contain at least one transgene capable of expressing a target pre-mRNA molecule. The invention provides methods, based on utilization of the transgenic animals, for assessing the specificity and efficiency of a pre-trans-splicing molecule (PTM) designed to interact with a target pre-mRNA and mediate a trans-splicing reaction resulting in the generation of a novel chimeric RNA molecule. The present invention further relates to the transgenic expression of PTM molecules in animals to determine gene function, i.e, functional genetics. The present invention is based on the successful generation of a transgenic animal expressing a target pre-mRNA and, moreover, the use of that animal to detect accurate in vivo trans-splicing reactions in the presence of a PTM.
摘要翻译：本发明涉及用于体内测试剪接体介导的RNA反式剪接反应的动物模型系统的开发。本发明提供了转基因动物，以及用于产生这样的动物的方法，其被遗传工程化以表达用作转拼反应的底物的靶前体信使RNA分子（靶前体mRNA）。具体地，转基因动物含有能够表达靶mRNA前分子的至少一个转基因。本发明提供了基于转基因动物利用的方法，用于评估设计成与靶前体mRNA相互作用的转录前分子（PTM）的特异性和效率，并介导导致产生转录的反式剪接反应的新型嵌合RNA分子。本发明还涉及PTM分子在动物中的转基因表达以确定基因功能，即功能遗传学。本发明基于成功生成表达靶mRNA的转基因动物，此外，该技术在PTM存在下使用该动物检测精确的体内转拼反应。

2. 发明申请

WO2007079141A3 METHOD OF IDENTIFYING COMPOUNDS USEFUL TO TREAT NEURONAL DEGENERATIVE DISEASES 审中-公开
标题翻译：鉴别化合物治疗神经性退行性疾病的方法
公开(公告)号：WO2007079141A3
公开(公告)日：2008-01-31
申请号：PCT/US2006049424
申请日：2006-12-28
申请人： UNIV IOWA RES FOUND , ENGELHARDT JOHN F , ZHOU WEIHONG , HARRAZ MAGED M , MARDEN JENNIFER J
发明人： ENGELHARDT JOHN F , ZHOU WEIHONG , HARRAZ MAGED M , MARDEN JENNIFER J
IPC分类号： G01N33/68 , A61K31/00 , A61K31/085 , A61K31/09
CPC分类号： A61K36/80 , A61K31/00 , A61K31/085 , A61K31/09 , C07K14/4706 , G01N33/6896 , G01N2333/90283 , G01N2333/914 , G01N2500/02 , G01N2800/28
摘要： Methods of identifying agents that inhibit ROS by altering the binding of a GTPase such as Rac to SOD, agents identified by the method, and methods of using compounds that inhibit ROS to inhibit or treat neuronal degenerative diseases, are provided.
摘要翻译：提供了通过改变GTP酶如Rac与SOD的结合来鉴定抑制ROS的试剂的方法，通过该方法鉴定的试剂以及使用抑制ROS抑制或治疗神经元退行性疾病的化合物的方法。

3. 发明申请

WO2007079141A9 METHOD OF IDENTIFYING COMPOUNDS USEFUL TO TREAT NEURONAL DEGENERATIVE DISEASES 审中-公开
标题翻译：鉴别化合物治疗神经性退行性疾病的方法
公开(公告)号：WO2007079141A9
公开(公告)日：2007-08-30
申请号：PCT/US2006049424
申请日：2006-12-28
申请人： UNIV IOWA RES FOUND , ENGELHARDT JOHN F , ZHOU WEIHONG , HARRAZ MAGED M , MARDEN JENNIFER J
发明人： ENGELHARDT JOHN F , ZHOU WEIHONG , HARRAZ MAGED M , MARDEN JENNIFER J
IPC分类号： C12Q1/42 , A61K36/00 , A61P25/28 , C07K14/00 , C12Q1/26
CPC分类号： A61K36/80 , A61K31/00 , A61K31/085 , A61K31/09 , C07K14/4706 , G01N33/6896 , G01N2333/90283 , G01N2333/914 , G01N2500/02 , G01N2800/28
摘要： Methods of identifying agents that inhibit ROS by altering the binding of a GTPase such as Rac to SOD, agents identified by the method, and methods of using compounds that inhibit ROS to inhibit or treat neuronal degenerative diseases, are provided.
摘要翻译：提供了通过改变GTP酶如Rac与SOD的结合来鉴定抑制ROS的试剂的方法，通过该方法鉴定的试剂以及使用抑制ROS抑制或治疗神经元退行性疾病的化合物的方法。

4. 发明申请

WO2004089423A3 PHARMICO-GENE THERAPHY OF EPITHELIAL SODIUM CHANNEL ASSOCIATED DISODERS 审中-公开
标题翻译：上一代钠通道相关物质的药代基因
公开(公告)号：WO2004089423A3
公开(公告)日：2005-04-21
申请号：PCT/US2004009950
申请日：2004-03-31
申请人： UNIV IOWA RES FOUND , ENGELHARDT JOHN F , ZHANG LIANG
发明人： ENGELHARDT JOHN F , ZHANG LIANG
IPC分类号： A61K48/00 , C12N15/86 , C12N15/861 , C12N15/864 , C12Q1/68 , C12Q1/70 , G01N33/50 , G01N33/68
CPC分类号： G01N33/502 , A61K48/00 , C12N15/86 , C12N2750/14143 , G01N33/5008 , G01N33/6872 , G01N33/6893
摘要： Agents and methods to alter epithelial sodium channel (EnaC) activity.
摘要翻译：改变上皮钠通道（EnaC）活性的药物和方法。

5. 发明申请

WO0075365A9 COMPOUNDS AND METHODS TO ENHANCE rAAV TRANSDUCTION 审中-公开
标题翻译：化合物和加强rAAV转导的方法
公开(公告)号：WO0075365A9
公开(公告)日：2002-05-02
申请号：PCT/US0015700
申请日：2000-06-08
申请人： UNIV IOWA RES FOUND , ENGELHARDT JOHN F , DUAN DONGSHENG
发明人： ENGELHARDT JOHN F , DUAN DONGSHENG
IPC分类号： C12N15/09 , A61K31/198 , A61K31/27 , A61K31/335 , A61K31/365 , A61K31/38 , A61K31/395 , A61K31/407 , A61K31/4184 , A61K31/4706 , A61K35/76 , A61K38/00 , A61K45/00 , A61K48/00 , A61P1/16 , A61P7/00 , A61P11/00 , A61P11/06 , A61P21/00 , A61P25/00 , A61P25/16 , A61P25/28 , A61P43/00 , C07K5/04 , C12N5/10 , C12N7/00 , C12Q1/02 , G01N33/50 , G01N33/569 , A61K38/55 , C12N15/86
CPC分类号： G01N33/5067 , A61K31/198 , A61K31/27 , A61K31/365 , A61K31/407 , A61K31/4184 , A61K31/4706 , A61K48/00 , C12N2799/025 , C12Q1/025 , G01N33/5008 , G01N33/502 , G01N33/5044 , G01N33/56983 , G01N2333/015 , G01N2333/075 , G01N2333/8139 , G01N2333/96466
摘要： Agents and methods to alter rAAV transduction are provided.
摘要翻译：提供了改变rAAV转导的药剂和方法。

6. 发明申请

WO2005111220A2 ADENO-ASSOCIATED VIRUS VECTORS WITH INTRAVECTOR HETEROLOGOUS TERMINAL PALINDROMIC SEQUENCES 审中-公开
标题翻译： ADENO相关病毒载体与入侵者异位终末期细菌序列
公开(公告)号：WO2005111220A2
公开(公告)日：2005-11-24
申请号：PCT/US2005015315
申请日：2005-05-02
申请人： UNIV IOWA RES FOUND , ENGELHARDT JOHN F , YAN ZIYING
发明人： ENGELHARDT JOHN F , YAN ZIYING
IPC分类号： A61K48/00 , C12N15/861 , C12N15/864
CPC分类号： C12N15/86 , A61K48/00 , A61K48/0008 , C12N2750/14143 , C12N2750/14145 , C12N2810/60
摘要： The invention provides recombinant AAV vectors, at least one of which has heterologous terminal palindromic sequences, and methods of using those vectors.
摘要翻译：本发明提供了重组AAV载体，其中至少一个具有异源末端回文序列，以及使用这些载体的方法。

7. 发明申请

WO2004090145A3 COMPOUNDS AND METHODS TO ENHANCE RAAV TRANSDUCTION 审中-公开
标题翻译：化合物和加强RAAV转移的方法
公开(公告)号：WO2004090145A3
公开(公告)日：2005-02-24
申请号：PCT/US2004010045
申请日：2004-03-31
申请人： UNIV IOWA RES FOUND , TARGETED GENETICS CORP , ENGELHARDT JOHN F , MUNSON KEITH L
发明人： ENGELHARDT JOHN F , MUNSON KEITH L
IPC分类号： A61K48/00 , C12N15/86 , C12N15/861 , C12N15/864 , C12Q1/68 , C12Q1/70 , G01N33/50 , G01N33/68
CPC分类号： G01N33/502 , A61K48/00 , C12N15/86 , C12N2750/14143 , G01N33/5008 , G01N33/6872 , G01N33/6893
摘要： Agents and methods to alter rAAV tranduction are provided.
摘要翻译：提供了改变rAAV转导的药剂和方法。

8. 发明申请

WO0125465A9 ADENO-ASSOCIATED VIRUSES AND USES THEREOF 审中-公开
标题翻译： ADENO相关病毒及其用途
公开(公告)号：WO0125465A9
公开(公告)日：2002-11-21
申请号：PCT/US0027863
申请日：2000-10-06
申请人： UNIV IOWA RES FOUND , ENGELHARDT JOHN F , DUAN DONGSHENG , YAN ZIYANG
发明人： ENGELHARDT JOHN F , DUAN DONGSHENG , YAN ZIYANG
IPC分类号： C12N15/09 , A61K35/76 , A61K38/00 , A61K48/00 , A61P43/00 , C12N5/10 , C12N7/01 , C12N15/864
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.
摘要翻译：本发明提供了分离和纯化的DNA分子，其包含腺伴随病毒的环状中间体的至少一个DNA区段，其生物活性亚单位或变体，该DNA区段赋予分离的DNA分子的增加的附加体稳定性，持久性或丰度在宿主细胞中。本发明还提供包含至少两个腺相关病毒载体的组合物。

9. 发明申请

WO2007127464A3 METHODS AND COMPOUNDS TO MODULATE PARVOVIRUS TRANSDUCTION OF MAMMALIAN CELLS OR TO ALTER VIRUS INFECTION, METHOD TO IDENTIFY A VIRAL RECEPTOR OR CO-RECEPTOR 审中-公开
标题翻译：用于调节哺乳动物细胞的寄生虫转移或改变病毒感染的方法和化合物，鉴定病毒受体或共同受体的方法
公开(公告)号：WO2007127464A3
公开(公告)日：2008-01-31
申请号：PCT/US2007010434
申请日：2007-04-27
申请人： UNIV IOWA RES FOUND , ENGELHARDT JOHN F , ZHANG LIANG
发明人： ENGELHARDT JOHN F , ZHANG LIANG
IPC分类号： G01N33/50 , C12N15/864 , G01N33/566 , G01N33/573
CPC分类号： G01N33/573 , A01K2217/075 , A01K2227/105 , A01K2267/0337 , C12N9/0036 , G01N33/502 , G01N33/566 , G01N2333/015 , G01N2333/075 , G01N2333/90209
摘要： The invention provides a method to identify an agent that alters parvovirus transduction of mammalian cells. Also provided is a method to enhance transgene expression in a mammalian cell, as well as a method to identify an agent that alters NADPH oxidase activity in parvovirus transduced mammalian cells.
摘要翻译：本发明提供了鉴定改变哺乳动物细胞的细小病毒转导的药剂的方法。还提供了增强哺乳动物细胞中转基因表达的方法，以及鉴定改变细小病毒转导的哺乳动物细胞中NADPH氧化酶活性的试剂的方法。

10. 发明申请

WO0075365A2 COMPOUNDS AND METHODS TO ENHANCE RAAV TRANSDUCTION 审中-公开
标题翻译：化合物和加强RAAV转移的方法
公开(公告)号：WO0075365A2
公开(公告)日：2000-12-14
申请号：PCT/US0015700
申请日：2000-06-08
申请人： UNIV IOWA RES FOUND , ENGELHARDT JOHN F , DUAN DONGSHENG
发明人： ENGELHARDT JOHN F , DUAN DONGSHENG
IPC分类号： C12N15/09 , A61K31/198 , A61K31/27 , A61K31/335 , A61K31/365 , A61K31/38 , A61K31/395 , A61K31/407 , A61K31/4184 , A61K31/4706 , A61K35/76 , A61K38/00 , A61K45/00 , A61K48/00 , A61P1/16 , A61P7/00 , A61P11/00 , A61P11/06 , A61P21/00 , A61P25/00 , A61P25/16 , A61P25/28 , A61P43/00 , C07K5/04 , C12N5/10 , C12N7/00 , C12Q1/02 , G01N33/50 , G01N33/569 , C12Q1/00
CPC分类号： G01N33/5067 , A61K31/198 , A61K31/27 , A61K31/365 , A61K31/407 , A61K31/4184 , A61K31/4706 , A61K48/00 , C12N2799/025 , C12Q1/025 , G01N33/5008 , G01N33/502 , G01N33/5044 , G01N33/56983 , G01N2333/015 , G01N2333/075 , G01N2333/8139 , G01N2333/96466
摘要： Agents and methods to alter rAAV transduction are provided.
摘要翻译：提供了改变rAAV转导的药剂和方法。

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