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公开(公告)号：US20130111615A1
公开(公告)日：2013-05-02
申请号：US13585517
申请日：2012-08-14
申请人： Katalin Kariko , Drew Weissman
发明人： Katalin Kariko , Drew Weissman
IPC分类号： C07H21/02
CPC分类号： A61K48/0066 , A61K38/177 , A61K38/1816 , A61K38/50 , A61K48/00 , A61K48/0041 , A61K48/005 , A61K48/0075 , C07H21/02 , C07K14/47 , C07K14/4712 , C07K14/505 , C12N15/117 , C12N15/67 , C12N15/85 , C12N2310/17 , C12N2310/33 , C12N2310/3341 , C12N2800/95 , C12Q1/68 , C12Y305/04004
摘要： This invention provides RNA, oligoribonucleotide, and polyribonucleotide molecules comprising pseudouridine or a modified nucleoside, gene therapy vectors comprising same, methods of synthesizing same, and methods for gene replacement, gene therapy, gene transcription silencing, and the delivery of therapeutic proteins to tissue in vivo, comprising the molecules. The present invention also provides methods of reducing the immunogenicity of RNA, oligoribonucleotide, and polyribonucleotide molecules.
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公开(公告)号：US20090286852A1
公开(公告)日：2009-11-19
申请号：US11990646
申请日：2006-08-21
申请人： Katalin Kariko , Drew Weissman
发明人： Katalin Kariko , Drew Weissman
IPC分类号： A61K31/713 , C07H21/02 , C12N15/00 , C12N15/63 , A61K48/00 , C12P19/34 , C07K5/00 , A61P7/06 , A61Q7/00
CPC分类号： A61K48/0066 , A61K38/177 , A61K38/1816 , A61K38/50 , A61K48/00 , A61K48/0041 , A61K48/005 , A61K48/0075 , C07H21/02 , C07K14/47 , C07K14/4712 , C07K14/505 , C12N15/117 , C12N15/67 , C12N15/85 , C12N2310/17 , C12N2310/33 , C12N2310/3341 , C12N2800/95 , C12Q1/68 , C12Y305/04004
摘要： This invention provides RNA, oligoribonucleotide, and polyribonucleotide molecules comprising pseudouridine or a modified nucleoside, gene therapy vectors comprising same, methods of synthesizing same, and methods for gene replacement, gene therapy, gene transcription silencing, and the delivery of therapeutic proteins to tissue in vivo, comprising the molecules. The present invention also provides methods of reducing the immunogenicity of RNA, oligoribonucleotide, and polyribonucleotide molecules.
摘要翻译： 本发明提供了包含假尿苷或修饰的核苷的RNA，寡核糖核苷酸和多核糖核苷酸分子，包含其的基因治疗载体，其合成方法，以及用于基因替换，基因治疗，基因转录沉默和将治疗性蛋白递送至组织的方法 包括分子。 本发明还提供降低RNA，寡核糖核苷酸和多核糖核苷酸分子的免疫原性的方法。
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公开(公告)号：US08691966B2
公开(公告)日：2014-04-08
申请号：US13585517
申请日：2012-08-14
申请人： Katalin Kariko , Drew Weissman
发明人： Katalin Kariko , Drew Weissman
CPC分类号： A61K48/0066 , A61K38/177 , A61K38/1816 , A61K38/50 , A61K48/00 , A61K48/0041 , A61K48/005 , A61K48/0075 , C07H21/02 , C07K14/47 , C07K14/4712 , C07K14/505 , C12N15/117 , C12N15/67 , C12N15/85 , C12N2310/17 , C12N2310/33 , C12N2310/3341 , C12N2800/95 , C12Q1/68 , C12Y305/04004
摘要： This invention provides RNA, oligoribonucleotide, and polyribonucleotide molecules comprising pseudouridine or a modified nucleoside, gene therapy vectors comprising same, methods of synthesizing same, and methods for gene replacement, gene therapy, gene transcription silencing, and the delivery of therapeutic proteins to tissue in vivo, comprising the molecules. The present invention also provides methods of reducing the immunogenicity of RNA, oligoribonucleotide, and polyribonucleotide molecules.
摘要翻译： 本发明提供了包含假尿苷或修饰的核苷的RNA，寡核糖核苷酸和多核糖核苷酸分子，包含其的基因治疗载体，其合成方法，以及用于基因替换，基因治疗，基因转录沉默和将治疗性蛋白递送至组织的方法 包括分子。 本发明还提供降低RNA，寡核糖核苷酸和多核糖核苷酸分子的免疫原性的方法。
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4.发明申请RNA PREPARATIONS COMPRISING PURIFIED MODIFIED RNA FOR REPROGRAMMING CELLS 有权包含用于转染细胞的纯化的修饰的RNA的RNA制备公开(公告)号：US20110143397A1
公开(公告)日：2011-06-16
申请号：US12962468
申请日：2010-12-07
申请人： Katalin Kariko , Drew Weissman , Gary Dahl , Anthony Person , Judith Meis , Jerome Jendrisak
发明人： Katalin Kariko , Drew Weissman , Gary Dahl , Anthony Person , Judith Meis , Jerome Jendrisak
CPC分类号： C12N5/0602 , A61K48/0041 , A61K48/005 , A61K48/0075 , C07K14/47 , C07K14/4712 , C07K14/505 , C12N5/0696 , C12N15/117 , C12N15/67 , C12N2310/17 , C12N2310/33 , C12N2310/3341 , C12N2501/40
摘要： The present invention provides compositions and methods for reprogramming somatic cells using purified RNA preparations comprising single-strand mRNA encoding an iPS cell induction factor. The purified RNA preparations are preferably substantially free of RNA contaminant molecules that: i) would activate an immune response in the somatic cells, ii) would decrease expression of the single-stranded mRNA in the somatic cells, and/or iii) active RNA sensors in the somatic cells. In certain embodiments, the purified RNA preparations are substantially free of partial mRNAs, double-stranded RNAs, un-capped RNA molecules, and/or single-stranded run-on mRNAs.
摘要翻译： 本发明提供使用包含编码iPS细胞诱导因子的单链mRNA的纯化RNA制备物重编程体细胞的组合物和方法。 纯化的RNA制剂优选基本上不含RNA污染物分子，其可以：i）激活体细胞中的免疫应答，ii）降低体细胞中单链mRNA的表达，和/或iii）活性RNA传感器 在体细胞中。 在某些实施方案中，纯化的RNA制剂基本上不含部分mRNA，双链RNA，未封端的RNA分子和/或单链连续的mRNA。
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公开(公告)号：US09012219B2
公开(公告)日：2015-04-21
申请号：US12962468
申请日：2010-12-07
申请人： Katalin Kariko , Drew Weissman , Gary Dahl , Anthony Person , Judith Meis , Jerome Jendrisak
发明人： Katalin Kariko , Drew Weissman , Gary Dahl , Anthony Person , Judith Meis , Jerome Jendrisak
IPC分类号： C12N5/00 , C12N5/02 , C12Q1/68 , A61K48/00 , C07K14/47 , C07K14/505 , C12N15/117 , C12N15/67
CPC分类号： C12N5/0602 , A61K48/0041 , A61K48/005 , A61K48/0075 , C07K14/47 , C07K14/4712 , C07K14/505 , C12N5/0696 , C12N15/117 , C12N15/67 , C12N2310/17 , C12N2310/33 , C12N2310/3341 , C12N2501/40
摘要： The present invention provides compositions and methods for reprogramming somatic cells using purified RNA preparations comprising single-strand mRNA encoding an iPS cell induction factor. The purified RNA preparations are preferably substantially free of RNA contaminant molecules that: i) would activate an immune response in the somatic cells, ii) would decrease expression of the single-stranded mRNA in the somatic cells, and/or iii) active RNA sensors in the somatic cells. In certain embodiments, the purified RNA preparations are substantially free of partial mRNAs, double-stranded RNAs, un-capped RNA molecules, and/or single-stranded run-on mRNAs.
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公开(公告)号：US08278036B2
公开(公告)日：2012-10-02
申请号：US11990646
申请日：2006-08-21
申请人： Katalin Kariko , Drew Weissman
发明人： Katalin Kariko , Drew Weissman
IPC分类号： C12Q1/68
CPC分类号： A61K48/0066 , A61K38/177 , A61K38/1816 , A61K38/50 , A61K48/00 , A61K48/0041 , A61K48/005 , A61K48/0075 , C07H21/02 , C07K14/47 , C07K14/4712 , C07K14/505 , C12N15/117 , C12N15/67 , C12N15/85 , C12N2310/17 , C12N2310/33 , C12N2310/3341 , C12N2800/95 , C12Q1/68 , C12Y305/04004
摘要： This invention provides RNA, oligoribonucleotide, and polyribonucleotide molecules comprising pseudouridine or a modified nucleoside, gene therapy vectors comprising same, methods of synthesizing same, and methods for gene replacement, gene therapy, gene transcription silencing, and the delivery of therapeutic proteins to tissue in vivo, comprising the molecules. The present invention also provides methods of reducing the immunogenicity of RNA, oligoribonucleotide, and polyribonucleotide molecules.
摘要翻译： 本发明提供了包含假尿苷或修饰的核苷的RNA，寡核糖核苷酸和多核糖核苷酸分子，包含其的基因治疗载体，其合成方法，以及用于基因替换，基因治疗，基因转录沉默和将治疗性蛋白递送至组织的方法 包括分子。 本发明还提供降低RNA，寡核糖核苷酸和多核糖核苷酸分子的免疫原性的方法。
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