专利快速检索-快速检索全球专利，免费商用专利数据库-IPRDB

1. 发明专利

CA2485341A1 USE OF SPLICEOSOME MEDIATED RNA TRANS-SPLICING TO CONFER CELL SELECTIVE REPLICATION TO ADENOVIRUSES 未知
公开(公告)号：CA2485341A1
公开(公告)日：2004-06-17
申请号：CA2485341
申请日：2003-05-08
申请人： INTRONN INC
发明人： OTTO EDWARD
IPC分类号： C12N15/09 , A61K48/00 , C12N5/10 , C12N7/00 , C12N15/861 , C12N7/01 , A61K35/76 , A61P35/00 , C12N15/87
摘要： The present invention provides methods and compositions for conferring tumor selective cell death on cancer cells expressing specific target precursor messenger RNA molecules (cancer cell selective target pre-mRNAs). The compositions of the invention include conditionally replicative adenoviruses that have been genetically engineered to express one or more pre-trans- splicing molecules (PTMs) designed to interact with one or more cancer cell target pre-mRNA and mediate a trans-splicing reaction resulting in the generation of novel chimeric RNA molecules (chimeric RNA) capable of encodin g adenovirus specific protein(s). Adenovirus specific proteins include those proteins complementing an essential activity necessary for replication of a defective adenovirus. The methods and compositions of the invention may be used to target a lytic adenovirus infection to cancer cells thereby providin g a method for selective destruction of cancer cells. In addition, the adenoviruses of the invention may be engineered to encode PTMs designed to interact with target pre-mRNAs encoded by infectious agents within a cell, thereby targeting selective destruction of cells infected with such agents.

2. 发明专利

AU2003285009A1 SCREENING METHODS FOR IDENTIFICATION OF EFFICIENT PRE-TRANS-SPLICING MOLECULES 未知
公开(公告)号：AU2003285009A1
公开(公告)日：2004-05-13
申请号：AU2003285009
申请日：2003-10-23
申请人： INTRONN INC
发明人： YANG YANPING , MITCHELL LLOYD G , PUTTARAJU MADAIAH , GARCIA-BLANCO MARIANO , OTTO EDWARD
IPC分类号： C07H21/04 , C12Q1/68 , G01N20060101 , G01N33/50 , G01N33/53
摘要： The present invention provides methods and compositions for rapid high capacity functional screening to identify optimal pre-trans-splicing molecules (PTMs). The compositions of the invention include PTM expression libraries capable of encoding candidate PTMs designed to interact with a target precursor messenger RNA molecule (target pre-mRNA) and mediate a trans-splicing reaction resulting in the generation of a novel chimeric RNA molecule (chimeric RNA). The candidate PTMs of the invention encode a portion of a first reporter molecule and may encode one or more other reporter molecules, which can be used to select for cells expressing optimal PTMs (efficient and specific). The compositions of the invention also include cells that express a target pre-mRNA encoding the remaining portion of the first reporter molecule. The screening methods of the invention encompass (i) contacting a PTM expression library with cells expressing a target pre-mRNA under conditions in which a trans-splicing reaction will occur in the presence of an optimal PTM (expressed by the library vector) resulting in the formation of a chimeric repaired RNA molecule capable of encoding at least one reporter molecule; (ii) selecting for cells expressing the repaired reporter molecule wherein expression of the reporter molecule indicates the presence of an optimal PTM in the selected cell; and (iii) identifying the optimal PTM expressed in the selected cell(s). The additional reporter molecule(s) can be used to assess both specific and non-specific trans-splicing, as well direct PTM expression.

3. 发明申请

WO2005023990A2 TRANS-SPLICING MEDIATED PHOTODYNAMIC THERAPY 审中-公开
标题翻译：转移介导的光电疗法
公开(公告)号：WO2005023990A2
公开(公告)日：2005-03-17
申请号：PCT/US2004029022
申请日：2004-09-07
申请人： INTRONN INC , MITCHELL LLOYD G , OTTO EDWARD , MERRIL CARL R
发明人： MITCHELL LLOYD G , OTTO EDWARD , MERRIL CARL R
IPC分类号： C12N15/09 , A61K31/7105 , A61K31/711 , A61K48/00 , C07H21/02 , C07H21/04 , C12N20060101 , C12N1/15 , C12N1/19 , C12N1/21 , C12N5/02 , C12N5/10 , C12N15/10 , C12N15/113 , C12N15/62 , C12N15/63 , C12N15/64 , C12N15/85 , C12N15/86 , C12N15/861 , C12P21/02 , C12Q1/02 , C12Q1/68 , C12N
CPC分类号： C12N15/10 , C12N15/1027 , C12N15/1086 , C12N15/111 , C12N15/113 , C12N2310/11 , C12N2310/111 , C12N2310/3519 , C12N2320/33 , C12N2840/445
摘要： The present invention provides methods and compositions for conferring selective death on cells expressing a specific target precursor messenger RNA (selective target pre-mRNA). The compositions of the invention include pre-trans-splicing molecules (PTMs) designed to interact with a target precursor messenger RNA molecule (target pre-mRMA) expressed within a cell and mediate a trans-splicing reaction resulting in the generation of a novel chimeric mRNA molecule (chimeric mRNA) capable of encoding a light producing protein or enzyme. Cell death is further mediated by the presence of a photosensitizer which upon photoactivation produces cytotoxicity.
摘要翻译：本发明提供了赋予表达特异性靶标前体信使RNA（选择性靶前体mRNA）的细胞上选择性死亡的方法和组合物。本发明的组合物包括设计成与在细胞内表达的靶前体信使RNA分子（靶前mRMA）相互作用的转录前分子（PTM），并介导反剪接反应，导致产生新的嵌合能够编码产生光的蛋白质或酶的mRNA分子（嵌合mRNA）。细胞死亡进一步由光敏剂的存在介导，光敏剂在光活化时产生细胞毒性。

4. 发明申请

WO2005070023A2 EXPRESSION OF APOA-1 AND VARIANTS THEREOF USING SPLICEOSOME MEDIATED RNA TRANS-SPLICING 审中-公开
标题翻译： APOA-1的表达及其使用SPLICEOSOME介导的RNA转移分离的变体
公开(公告)号：WO2005070023A2
公开(公告)日：2005-08-04
申请号：PCT/US2005002392
申请日：2005-01-21
申请人： INTRONN INC , PUTTARAJU MADAIAH , OTTO EDWARD , GARCIA-BLANCO MARIANO A , MCGARRITY GERARD J , TEMPLE GARY F , MITCHELL LLOYD G
发明人： PUTTARAJU MADAIAH , OTTO EDWARD , GARCIA-BLANCO MARIANO A , MCGARRITY GERARD J , TEMPLE GARY F , MITCHELL LLOYD G
IPC分类号： C07K14/775 , C12N15/11 , C12N15/113
CPC分类号： C07K14/775 , C12N15/111 , C12N15/113 , C12N2310/11 , C12N2310/3519 , C12N2320/33
摘要： The present invention provides methods and compositions for generating novel nucleic acid molecules through targeted spliceosome mediated RNA trans-splicing that result in expression of an apoA-1 variant, the preferred embodiment referred to herein as the apoA-1 Milano variant. The compositions of the invention include pre-trans-splicing molecules (PTMs) designed to interact with a target precursor messenger RNA molecule (target pre-mRNA) and mediate a trans-splicing reaction resulting in the generation of a novel chimeric RNA molecule (chimeric RNA) capable of encoding the apoA-1 Milano variant. The expression of this variant protein results in protection against vascular disorders resulting from plaque build up, i.e., strokes and heart attacks. In particular, the PTMs of the presént invention include those genetically engineered to interact with the apoA-1 target premRNA so as to result in expression of the apoA-1 Milano variant. In addition, the PTMs of the invention include those genetically engineered to interact with the apoB or albumin or other specific target pre-mRNAs so as to result in expression of an apoB/apoA-1 and/or alb/apoA-1 wild type or Milano fusion protein thereby reducing apoB expression and simultaneously produce ApoA-1 function.
摘要翻译：本发明提供了通过靶向剪接体介导的RNA转接产生新的核酸分子的方法和组合物，其导致apoA-1变体（本文中称为apoA-1 Milano变体）的优选实施方案的表达。本发明的组合物包括被设计成与靶前体信使RNA分子（靶前mRNA）相互作用并且介导导致产生新的嵌合RNA分子（嵌合体）的反式剪接反应的预转录分子（PTM） RNA）能够编码apoA-1 Milano变体。该变体蛋白质的表达导致保护免受斑块积聚引起的血管疾病，即中风和心脏病发作。特别地，预发明的PTM包括经遗传工程改造以与apoA-1靶preRNA相互作用以产生载脂蛋白-A 1米兰变体的表达的那些。此外，本发明的PTM包括经遗传工程改造以与载脂蛋白B或白蛋白或其它特异性靶前体mRNA相互作用以产生apoB / apoA-1和/或alb / apoA-1野生型或米诺融合蛋白从而降低apoB表达并同时产生ApoA-1功能。

5. 发明专利

CA2553828A1 EXPRESSION OF APOA-1 AND VARIANTS THEREOF USING SPLICEOSOME MEDIATED RNA TRANS-SPLICING 未知
公开(公告)号：CA2553828A1
公开(公告)日：2005-08-04
申请号：CA2553828
申请日：2005-01-21
申请人： INTRONN INC
发明人： GARCIA-BLANCO MARIANO A , MCGARRITY GERARD J , OTTO EDWARD , PUTTARAJU MADAIAH , MITCHELL LLOYD G , TEMPLE GARY F
IPC分类号： C07H21/02 , C07K14/775 , C12N5/10 , C12N15/09 , C12N15/11 , C12N15/113 , C12P19/34
摘要： The present invention provides methods and compositions for generating novel nucleic acid molecules through targeted spliceosome mediated RNA trans- splicing that result in expression of an apoA-1 variant, the preferred embodiment referred to herein as the apoA-1 Milano variant. The compositions of the invention include pre-trans-splicing molecules (PTMs) designed to interact with a target precursor messenger RNA molecule (target pre-mRNA) an d mediate a trans~-splicing reaction resulting in the generation of a novel chimeric RNA molecule (chimeric RNA) capable of encoding the apoA-1 Milano variant. The expression of this variant protein results in protection agains t vascular disorders resulting from plaque build up, i.e., strokes and heart attacks. In particular, the PTMs of the presént invention include those genetically engineered to interact with the apoA-1 target pre~mRNA so as to result in expression of the apoA-1 Milano variant. In addition, the PTMs of the invention include those genetically engineered to interact with the apoB or albumin or other specific target pre-mRNAs so as to result in expression of an apoB/apoA-1 and/or alb/apoA-1 wild type or Milano fusion protein thereby reducing apoB expression and simultaneously produce ApoA-1 function.

6. 发明专利

CA2503247A1 SCREENING METHODS FOR IDENTIFICATION OF EFFICIENT PRE-TRANS-SPLICING MOLECULES 未知
公开(公告)号：CA2503247A1
公开(公告)日：2004-05-06
申请号：CA2503247
申请日：2003-10-23
申请人： INTRONN INC
发明人： MITCHELL LLOYD G , YANG YANPING , PUTTARAJU MADAIAH , GARCIA-BLANCO MARIANO , OTTO EDWARD
IPC分类号： C07H21/04 , C12Q1/68 , G01N20060101 , G01N33/50 , G01N33/53
摘要： The present invention provides methods and compositions for rapid high capacity functional screening to identify optimal pre-trans-splicing molecul es (PTMs). The compositions of the invention include PTM expression libraries capable of encoding candidate PTMs designed to interact with a target precursor messenger RNA molecule (target pre-mRNA) and mediate a trans- splicing reaction resulting in the generation of a novel chimeric RNA molecu le (chimeric RNA). The candidate PTMs of the invention encode a portion of a first reporter molecule and may encode one or more other reporter molecules, which can be used to select for cells expressing optimal PTMs (efficient and specific). The compositions of the invention also include cells that express a target pre-mRNA encoding the remaining portion of the first reporter molecul e. The screening methods of the invention encompass (i) contacting a PTM expression library with cells expressing a target pre-mRNA under conditions in which a trans-splicing reaction will occur in the presence of an optimal PTM (expressed by the library vector) resulting in the formation of a chimeric repaired RNA molecule capable of encoding at least one reporter molecule; (i i) selecting for cells expressing the repaired reporter molecule wherein expression of the reporter molecule indicates the presence of an optimal PTM in the selected cell; and (iii) identifying the optimal PTM expressed in the selected cell(s). The additional reporter molecule(s) can be used to assess both specific and non-specific trans-splicing, as well direct PTM expression .

7. 发明申请

WO2004050680A2 USE OF SPLICEOSOME MEDIATED RNA TRANS-SPLICING TO CONFER CELL SELECTIVE REPLICATION TO ADENOVIRUSES 审中-公开
标题翻译：使用SPLICEOSOME介导的RNA转分离技术将细胞选择性复制给腺病毒
公开(公告)号：WO2004050680A2
公开(公告)日：2004-06-17
申请号：PCT/US0314804
申请日：2003-05-08
申请人： INTRONN INC
发明人： OTTO EDWARD
IPC分类号： C12N15/09 , A61K48/00 , C12N5/10 , C12N7/00 , C12N15/861 , C07K
CPC分类号： C12N15/86 , A61K48/00 , C12N2710/10343 , C12N2710/10345 , C12N2840/445
摘要： The present invention provides methods and compositions for conferring tumor selective cell death on cancer cells expressing specific target precursor messenger RNA molecules (cancer cell selective target pre-mRNAs). The compositions of the invention include conditionally replicative adenoviruses that have been genetically engineered to express one or more pre-trans-splicing molecules (PTMs) designed to interact with one or more cancer cell target pre-mRNA and mediate a trans-splicing reaction resulting in the generation of novel chimeric RNA molecules (chimeric RNA) capable of encoding adenovirus specific protein(s). Adenovirus specific proteins include those proteins complementing an essential activity necessary for replication of a defective adenovirus. The methods and compositions of the invention may be used to target a lytic adenovirus infection to cancer cells thereby providing a method for selective destruction of cancer cells. In addition, the adenoviruses of the invention may be engineered to encode PTMs designed to interact with target pre-mRNAs encoded by infectious agents within a cell, thereby targeting selective destruction of cells infected with such agents.
摘要翻译：本发明提供了在表达特异性靶标前体信使RNA分子（癌细胞选择性靶mRNA前体）的癌细胞上赋予肿瘤选择性细胞死亡的方法和组合物。本发明的组合物包括有条件复制的腺病毒，其被遗传工程化以表达设计成与一种或多种癌细胞靶前体mRNA相互作用的一种或多种预转录分子（PTM）并介导反剪接反应，生成能够编码腺病毒特异性蛋白质的新型嵌合RNA分子（嵌合RNA）。腺病毒特异性蛋白质包括补充有缺陷型腺病毒复制必需活性的蛋白质。本发明的方法和组合物可用于将溶解性腺病毒感染靶向癌细胞，从而提供选择性破坏癌细胞的方法。此外，本发明的腺病毒可以被设计成编码设计成与细胞内的感染因子编码的靶前mRNA相互作用的PTM，从而靶向用这种药剂感染的细胞的选择性破坏。

8. 发明申请

WO2004038380A2 SCREENING METHODS FOR IDENTIFICATION OF EFFICIENT PRE-TRANS-SPLICING MOLECULES 审中-公开
标题翻译：用于识别有效预分离分子的筛选方法
公开(公告)号：WO2004038380A2
公开(公告)日：2004-05-06
申请号：PCT/US0334102
申请日：2003-10-23
申请人： INTRONN INC , MITCHELL LLOYD G , PUTTARAJU MADAIAH , GARCIA-BLANCO MARIANO , OTTO EDWARD , YANG YANPING
发明人： MITCHELL LLOYD G , PUTTARAJU MADAIAH , GARCIA-BLANCO MARIANO , OTTO EDWARD , YANG YANPING
IPC分类号： C07H21/04 , C12Q1/68 , G01N20060101 , G01N33/50 , G01N33/53 , G01N
CPC分类号： C12N15/1027 , C12N15/1086 , G01N33/5023
摘要： The present invention provides methods and compositions for rapid high capacity functional screening to identify optimal pre-trans-splicing molecules (PTMs). The compositions of the invention include PTM expression libraries capable of encoding candidate PTMs designed to interact with a target precursor messenger RNA molecule (target pre-mRNA) and mediate a trans-splicing reaction resulting in the generation of a novel chimeric RNA molecule (chimeric RNA). The candidate PTMs of the invention encode a portion of a first reporter molecule and may encode one or more other reporter molecules, which can be used to select for cells expressing optimal PTMs (efficient and specific). The compositions of the invention also include cells that express a target pre-mRNA encoding the remaining portion of the first reporter molecule. The screening methods of the invention encompass (i) contacting a PTM expression library with cells expressing a target pre-mRNA under conditions in which a trans-splicing reaction will occur in the presence of an optimal PTM (expressed by the library vector) resulting in the formation of a chimeric repaired RNA molecule capable of encoding at least one reporter molecule; (ii) selecting for cells expressing the repaired reporter molecule wherein expression of the reporter molecule indicates the presence of an optimal PTM in the selected cell; and (iii) identifying the optimal PTM expressed in the selected cell(s). The additional reporter molecule(s) can be used to assess both specific and non-specific trans-splicing, as well direct PTM expression.
摘要翻译：本发明提供用于快速高容量功能筛选以鉴定最佳预转录分子（PTM）的方法和组合物。本发明的组合物包括能够编码设计成与靶前体信使RNA分子（靶前体mRNA）相互作用的候选PTM的PTM表达文库，并介导产生新型嵌合RNA分子的转拼反应（嵌合RNA ）。本发明的候选PTM编码第一报告分子的一部分并且可以编码一个或多个其它报告分子，其可用于选择表达最佳PTM（有效和特异性）的细胞。本发明的组合物还包括表达编码第一报告分子的剩余部分的靶mRNA的细胞。本发明的筛选方法包括（i）在最优PTM（由文库载体表达）存在下在其中将发生转拼反应的条件下使PTM表达文库与表达靶mRNA的细胞接触，导致形成能够编码至少一个报告分子的嵌合修复的RNA分子; （ii）选择表达修复的报告分子的细胞，其中报道分子的表达指示所选细胞中存在最佳PTM; 和（iii）鉴定在所选择的细胞中表达的最佳PTM。另外的报告分子可用于评估特异性和非特异性转拼，以及直接PTM表达。

9. 发明公开

EP1504016A4 USE OF SPLICEOSOME MEDIATED RNA TRANS-SPLICING TO CONFER CELL SELECTIVE REPLICATION TO ADENOVIRUSES 审中-公开
标题翻译： USE剪接体介导的RNA反式剪接，以腺病毒细胞选择REPLICATION贷款
公开(公告)号：EP1504016A4
公开(公告)日：2006-11-22
申请号：EP03810034
申请日：2003-05-08
申请人： INTRONN INC
发明人： OTTO EDWARD
IPC分类号： C12N15/09 , C12N15/861 , A61K35/76 , A61K48/00 , C12N5/10 , C12N7/00
CPC分类号： C12N15/86 , A61K48/00 , C12N2710/10343 , C12N2710/10345 , C12N2840/445

10. 发明公开

EP1579004A4 SCREENING METHODS FOR IDENTIFICATION OF EFFICIENT PRE-TRANS-SPLICING MOLECULES 有权
标题翻译：筛选方法，确定有效的PRE-TRANS-SPLEISSMOLEKÜLE
公开(公告)号：EP1579004A4
公开(公告)日：2006-06-28
申请号：EP03779324
申请日：2003-10-23
申请人： INTRONN INC
发明人： MITCHELL LLOYD G , PUTTARAJU MADAIAH , GARCIA-BLANCO MARIANO , OTTO EDWARD , YANG YANPING
IPC分类号： C07H21/04 , C12Q1/68 , G01N20060101 , G01N33/50 , G01N33/53
CPC分类号： C12N15/1027 , C12N15/1086 , G01N33/5023
摘要： The present invention provides methods and compositions for rapid high capacity functional screening to identify optimal pre-trans-splicing molecules (PTMs). The compositions of the invention include PTM expression libraries capable of encoding candidate PTMs designed to interact with a target precursor messenger RNA molecule (target pre-mRNA) and mediate a trans-splicing reaction resulting in the generation of a novel chimeric RNA molecule (chimeric RNA). The candidate PTMs of the invention encode a portion of a first reporter molecule and may encode one or more other reporter molecules, which can be used to select for cells expressing optimal PTMs (efficient and specific). The compositions of the invention also include cells that express a target pre-mRNA encoding the remaining portion of the first reporter molecule. The screening methods of the invention encompass (i) contacting a PTM expression library with cells expressing a target pre-mRNA under conditions in which a trans-splicing reaction will occur in the presence of an optimal PTM (expressed by the library vector) resulting in the formation of a chimeric repaired RNA molecule capable of encoding at least one reporter molecule; (ii) selecting for cells expressing the repaired reporter molecule wherein expression of the reporter molecule indicates the presence of an optimal PTM in the selected cell; and (iii) identifying the optimal PTM expressed in the selected cell(s). The additional reporter molecule(s) can be used to assess both specific and non-specific trans-splicing, as well direct PTM expression.

你已经成功收藏专利！

检索式保存成功!

IPRDB

热门服务

关于我们

友情链接

联系方式