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1. 发明申请

WO2010105020A3 BACULOVIRUSES WITH ENHANCED VIRION PRODUCTION AND A METHOD FOR THE PRODUCTION OF BACULOVIRUSES 审中-公开
标题翻译：具有增强的病毒生产的细菌病毒和生产细菌病毒的方法
公开(公告)号：WO2010105020A3
公开(公告)日：2011-01-13
申请号：PCT/US2010026922
申请日：2010-03-11
申请人： THOMPSON BOYCE PLANT RES , MCCRAY PAUL , SINN PATRICK , BLISSARD GARY , UNIV IOWA RES FOUND
发明人： MCCRAY PAUL , SINN PATRICK , BLISSARD GARY
IPC分类号： C12N15/867 , C12N7/01 , C12N15/66
CPC分类号： C12N7/00 , C12N15/86 , C12N2710/14121 , C12N2710/14143 , C12N2710/14151
摘要： A method of co-expressing a portion of the VSV G protein gene or a truncated "stem" portion with GP64 and a retrovirus increases the titer of retroviral vectors. A truncated VSV G protein, preferably comprised of a small segment from the C-terminal portion of the ectodomain plus the transmembrane (TM) and cytoplasmic tail (CTD) domains of VSV G, co-expressed with retroviral vectors, enhances the production titers of the retroviral vectors. A preferred embodiment uses a VSV G construct that includes an N-terminal c-Myc epitope plus 42 amino acids from the C-terminal portion of the ectodomain, 20 amino acids from the predicted TM domain, and 29 amino acids from the predicted CTD of the VSV G protein.
摘要翻译：用GP64和逆转录病毒共表达VSV G蛋白基因的一部分或截短的“茎”部分的方法提高了逆转录病毒载体的滴度。截短的VSV G蛋白，优选包含来自胞外域的C-末端部分加VSV G的跨膜（TM）和细胞质尾（CTD）结构域的小片段，其与逆转录病毒载体共表达，增强了生物滴度逆转录病毒载体。优选实施方案使用VSV G构建体，其包含N-末端c-Myc表位加上来自胞外结构域的C末端部分的42个氨基酸，来自预测的TM结构域的20个氨基酸，以及来自预测的CTD的29个氨基酸 VSV G蛋白。

2. 发明申请

WO2004104032A2 METHODS AND COMPOSITIONS RELATED TO HIGH-TITER PSEUDOTYPED RETROVIRUSES 审中-公开
标题翻译：方法与组合物相关的高蛋白复制体病毒
公开(公告)号：WO2004104032A2
公开(公告)日：2004-12-02
申请号：PCT/US2004014897
申请日：2004-05-13
申请人： UNIV IOWA RES FOUND , MCCRAY PAUL , FAN HUNG , SINN PATRICK
发明人： MCCRAY PAUL , FAN HUNG , SINN PATRICK
IPC分类号： A61K48/00 , C12N7/00 , C12N15/867 , C07K14/155
CPC分类号： C12N15/86 , A61K48/00 , C12N7/00 , C12N2740/12043 , C12N2740/15021 , C12N2740/15043 , C12N2740/15045 , C12N2830/003 , C12N2840/44
摘要： The present invention concerns the methods and compositions related to pseudotyped viral vectors. Embodiments of the invention include pseudotyping expression cassettes that include nucleic acid elements for enhancing the titer of pseudotyped viral particles. Embodiments of the invention include novel methods and compositions related to making high titer pseudotyped retroviral vector compositions. A heterologous envelope glycoprotein is typically incorporated into the virus during the budding or virus production process. Certain embodiments of the invention include pseudotyped retroviral vectors comprising a heterologous envelope glycoprotein derived from a Jaagsiekte sheep retrovirus (JSRV env). Pseudotyped viruses or viral particles may have a modified host range that is influenced by the properties of the heterologous envelope glycoprotein. Thus, embodiments of the invention include improved methods and compositions related to pseudotyped viruses suitable for ex vivo and in vivo methods including gene transfer and other therapeutic and experimental methods.
摘要翻译：本发明涉及与假型病毒载体相关的方法和组合物。本发明的实施方案包括假型分型表达盒，其包括用于增强假型病毒颗粒滴度的核酸元件。本发明的实施方案包括与制备高滴度假型逆转录病毒载体组合物有关的新方法和组合物。在发芽或病毒生产过程中，异源包膜糖蛋白通常并入病毒。本发明的某些实施方案包括包含衍生自Jaagsiekte绵羊逆转录病毒（JSRV env）的异源包膜糖蛋白的假型逆转录病毒载体。伪型病毒或病毒颗粒可能具有受异源包膜糖蛋白特性影响的修饰的宿主范围。因此，本发明的实施方案包括与适用于离体和体内方法（包括基因转移和其他治疗和实验方法）的假型病毒相关的改进的方法和组合物。

3. 发明申请

WO2010105020A2 BACULOVIRUSES WITH ENHANCED VIRION PRODUCTION AND A METHOD FOR THE PRODUCTION OF BACULOVIRUSES 审中-公开
标题翻译：具有增强的病毒生产的细菌病毒和生产细菌病毒的方法
公开(公告)号：WO2010105020A2
公开(公告)日：2010-09-16
申请号：PCT/US2010/026922
申请日：2010-03-11
申请人： BOYCE THOMPSON INSTITUTE FOR PLANT RESEARCH , MCCRAY, Paul , SINN, Patrick , BLISSARD, Gary , UNIVERSITY OF IOWA RESEARCH FOUNDATION
发明人： MCCRAY, Paul , SINN, Patrick , BLISSARD, Gary
IPC分类号： C12N15/867 , C12N15/66 , C12N7/01
CPC分类号： C12N7/00 , C12N15/86 , C12N2710/14121 , C12N2710/14143 , C12N2710/14151
摘要： A method of co-expressing a portion of the VSV G protein gene or a truncated "stem" portion with GP64 and a retrovirus increases the titer of retroviral vectors. A truncated VSV G protein, preferably comprised of a small segment from the C-terminal portion of the ectodomain plus the transmembrane (TM) and cytoplasmic tail (CTD) domains of VSV G, co-expressed with retroviral vectors, enhances the production titers of the retroviral vectors. A preferred embodiment uses a VSV G construct that includes an N-terminal c-Myc epitope plus 42 amino acids from the C-terminal portion of the ectodomain, 20 amino acids from the predicted TM domain, and 29 amino acids from the predicted CTD of the VSV G protein.
摘要翻译：用GP64和逆转录病毒共表达VSV G蛋白基因的一部分或截短的“茎”部分的方法提高了逆转录病毒载体的滴度。截短的VSV G蛋白，优选包含来自胞外域的C-末端部分加VSV G的跨膜（TM）和细胞质尾（CTD）结构域的小片段，其与逆转录病毒载体共表达，增强了生物滴度逆转录病毒载体。优选实施方案使用VSV G构建体，其包含N-末端c-Myc表位加上来自胞外结构域的C末端部分的42个氨基酸，来自预测的TM结构域的20个氨基酸，以及来自预测的CTD的29个氨基酸 VSV G蛋白。

4. 发明申请

WO2006085987A9 RNA INTERFERENCE IN RESPIRATORY EPITHEIAL CELLS 审中-公开
标题翻译： RNA在呼吸细胞上皮细胞中的干扰
公开(公告)号：WO2006085987A9
公开(公告)日：2006-10-19
申请号：PCT/US2005024626
申请日：2005-07-11
申请人： UNIV IOWA RES FOUND , MCCRAY PAUL B , DAVIDSON BEVERLY L , FISCHER ANTHONY J , JIA HONG P , DONOVAN MAUREEN D , SINN PATRICK L , BEHLKE MARK AARON
发明人： MCCRAY PAUL B , DAVIDSON BEVERLY L , FISCHER ANTHONY J , JIA HONG P , DONOVAN MAUREEN D , SINN PATRICK L , BEHLKE MARK AARON
IPC分类号： C12N15/11 , C12N15/113
CPC分类号： C12N15/1131 , C12N15/111 , C12N15/1136 , C12N15/1137 , C12N2310/111 , C12N2310/14 , C12N2310/53 , C12N2320/31 , C12N2320/32
摘要： The present invention is directed to small interfering RNA molecules targeted against a gene of interest in respiratory epithelial cells, and methods of using these RNA molecules.
摘要翻译：本发明涉及针对呼吸道上皮细胞中感兴趣基因的小干扰RNA分子，以及使用这些RNA分子的方法。

5. 发明申请

WO2004104032A3 METHODS AND COMPOSITIONS RELATED TO HIGH-TITER PSEUDOTYPED RETROVIRUSES 审中-公开
公开(公告)号：WO2004104032A3
公开(公告)日：2004-12-02
申请号：PCT/US2004/014897
申请日：2004-05-13
申请人： THE UNIVERSITY OF IOWA RESEARCH FOUNDATION , THE REGENTS OF THE UNIVERSITY OF CALIFORNIA , MCCRAY, Paul , FAN, Hung , SINN, Patrick
发明人： MCCRAY, Paul , FAN, Hung , SINN, Patrick
IPC分类号： C12N15/867
摘要： The present invention concerns the methods and compositions related to pseudotyped viral vectors. Embodiments of the invention include pseudotyping expression cassettes that include nucleic acid elements for enhancing the titer of pseudotyped viral particles. Embodiments of the invention include novel methods and compositions related to making high titer pseudotyped retroviral vector compositions. A heterologous envelope glycoprotein is typically incorporated into the virus during the budding or virus production process. Certain embodiments of the invention include pseudotyped retroviral vectors comprising a heterologous envelope glycoprotein derived from a Jaagsiekte sheep retrovirus (JSRV env). Pseudotyped viruses or viral particles may have a modified host range that is influenced by the properties of the heterologous envelope glycoprotein. Thus, embodiments of the invention include improved methods and compositions related to pseudotyped viruses suitable for ex vivo and in vivo methods including gene transfer and other therapeutic and experimental methods.

6. 发明申请

WO2006085987A2 RNA INTERFERENCE IN RESPIRATORY EPITHEIAL CELLS 审中-公开
标题翻译：呼吸道上皮细胞中的RNA干扰
公开(公告)号：WO2006085987A2
公开(公告)日：2006-08-17
申请号：PCT/US2005/024626
申请日：2005-07-11
申请人： UNIVERSITY OF IOWA RESEARCH FOUNDATION , MCCRAY, Paul, B. , DAVIDSON, Beverly, L. , FISCHER, Anthony, J. , JIA, Hong, P. , DONOVAN, Maureen, D. , SINN, Patrick, L. , BEHLKE, Mark, Aaron
发明人： MCCRAY, Paul, B. , DAVIDSON, Beverly, L. , FISCHER, Anthony, J. , JIA, Hong, P. , DONOVAN, Maureen, D. , SINN, Patrick, L. , BEHLKE, Mark, Aaron
IPC分类号： C12N15/11 , C12N15/113
CPC分类号： C12N15/1131 , C12N15/111 , C12N15/1136 , C12N15/1137 , C12N2310/111 , C12N2310/14 , C12N2310/53 , C12N2320/31 , C12N2320/32
摘要： The present invention is directed to small interfering RNA molecules targeted against a gene of interest in respiratory epithelial cells, and methods of using these RNA molecules.
摘要翻译：本发明涉及针对呼吸上皮细胞中目的基因的小干扰RNA分子，以及使用这些RNA分子的方法。

7. 发明申请

WO2006078431A2 COMPOSITIONS AND METHODS RELATED TO MODIFIED RETROVIRAL VECTORS FOR RESTRICTED, SITE SPECIFIC INTEGRATION 审中-公开
标题翻译：受限制的，特定地点集成的修饰的逆转录病毒载体的组合物和方法
公开(公告)号：WO2006078431A2
公开(公告)日：2006-07-27
申请号：PCT/US2005/047378
申请日：2005-12-22
申请人： THE UNIVERSITY OF IOWA RESEARCH FOUNDATION , IOWA STATE UNIVERSITY RESEARCH FOUNDATION, INC. , McCRAY, Paul, B., Jr. , SINN, Patrick, L. , VOYTAS, Daniel, F. , DAI, Junbiao
发明人： McCRAY, Paul, B., Jr. , SINN, Patrick, L. , VOYTAS, Daniel, F. , DAI, Junbiao
CPC分类号： C07K14/005 , C07K2319/70 , C07K2319/71 , C07K2319/81 , C12N9/22 , C12N15/86 , C12N15/90 , C12N2740/15022 , C12N2740/15043 , C12N2740/16122 , C12N2740/16222
摘要： Embodiments of the invention include compositions comprising and methods utilizing a retroviral integrase complex comprising a recombinant integrase having a domain comprising a non-native protein binding site, and a DNA binding protein comprising a DNA binding domain and a peptide binding domain that binds the nonnative protein binding site of the recombinant integrase.
摘要翻译：本发明的实施方案包括组合物和方法，所述组合物包含和使用逆转录病毒整合酶复合物的方法，所述逆转录病毒整合酶复合物包含具有包含非天然蛋白质结合位点的结构域的重组整合酶和包含DNA结合结构域和肽结合结构域，其结合重组整合酶的非天然蛋白质结合位点。

8. 发明申请

WO2021222222A1 COMPOSITIONS AND METHODS FOR THE TREATMENT OF CYSTIC FIBROSIS 审中-公开
公开(公告)号：WO2021222222A1
公开(公告)日：2021-11-04
申请号：PCT/US2021/029365
申请日：2021-04-27
申请人： MCCRAY JR., Paul B. , SINN, Patrick , LOZA, Laura Marquez
发明人： MCCRAY JR., Paul B. , SINN, Patrick , LOZA, Laura Marquez
IPC分类号： C12N15/86 , A61K48/00 , C07K14/47
摘要： Provided herein are polynucleotides, lentiviral vectors, pharmaceutical compositions, and methods of making and using the same, e.g., for treatment of cystic fibrosis (CF).

9. 发明申请

WO2006085987A3 RNA INTERFERENCE IN RESPIRATORY EPITHEIAL CELLS 审中-公开
标题翻译：呼吸道上皮细胞中的RNA干扰
公开(公告)号：WO2006085987A3
公开(公告)日：2007-06-28
申请号：PCT/US2005024626
申请日：2005-07-11
申请人： UNIV IOWA RES FOUND , MCCRAY PAUL B , DAVIDSON BEVERLY L , FISCHER ANTHONY J , JIA HONG P , DONOVAN MAUREEN D , SINN PATRICK L , BEHLKE MARK AARON
发明人： MCCRAY PAUL B , DAVIDSON BEVERLY L , FISCHER ANTHONY J , JIA HONG P , DONOVAN MAUREEN D , SINN PATRICK L , BEHLKE MARK AARON
IPC分类号： A61K31/713 , A61P11/00 , A61P29/00 , C12N5/10 , C12N15/11 , C12N15/113
CPC分类号： C12N15/1131 , C12N15/111 , C12N15/1136 , C12N15/1137 , C12N2310/111 , C12N2310/14 , C12N2310/53 , C12N2320/31 , C12N2320/32
摘要： The present invention is directed to small interfering RNA molecules targeted against a gene of interest in respiratory epithelial cells, and methods of using these RNA molecules.
摘要翻译：本发明涉及针对呼吸上皮细胞中目的基因的小干扰RNA分子，以及使用这些RNA分子的方法。

10. 发明申请

WO2006078431A3 COMPOSITIONS AND METHODS RELATED TO MODIFIED RETROVIRAL VECTORS FOR RESTRICTED, SITE SPECIFIC INTEGRATION 审中-公开
标题翻译：用于限制性，现场特定整合的修改后退向量的组合物和方法
公开(公告)号：WO2006078431A3
公开(公告)日：2006-10-12
申请号：PCT/US2005047378
申请日：2005-12-22
申请人： UNIV IOWA RES FOUND , UNIV IOWA STATE RES FOUND INC , MCCRAY PAUL B JR , SINN PATRICK L , VOYTAS DANIEL F , DAI JUNBIAO
发明人： MCCRAY PAUL B JR , SINN PATRICK L , VOYTAS DANIEL F , DAI JUNBIAO
IPC分类号： C12N9/22 , C12N15/85 , C12N15/90
CPC分类号： C07K14/005 , C07K2319/70 , C07K2319/71 , C07K2319/81 , C12N9/22 , C12N15/86 , C12N15/90 , C12N2740/15022 , C12N2740/15043 , C12N2740/16122 , C12N2740/16222
摘要： Embodiments of the invention include compositions comprising and methods utilizing a retroviral integrase complex comprising a recombinant integrase having a domain comprising a non-native protein binding site, and a DNA binding protein comprising a DNA binding domain and a peptide binding domain that binds the nonnative protein binding site of the recombinant integrase.
摘要翻译：本发明的实施方案包括使用包含具有包含非天然蛋白结合位点的结构域的重组整合酶的逆转录病毒整合酶复合物的组合物和包含结合非天然蛋白质的DNA结合结构域和肽结合结构域的DNA结合蛋白重组整合酶的结合位点。

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