公开(公告)号：US20090253206A1

公开(公告)日：2009-10-08

申请号：US12440416

申请日：2007-09-13

申请人： Gregor Siebenkotten ,  Bodo Ortmann

发明人： Gregor Siebenkotten ,  Bodo Ortmann

IPC分类号： C12N15/87 ,  C07K2/00 ,  C07C57/00 ,  C07K7/00 ,  C07K14/00

CPC分类号： C12N15/87 ,  C07K2319/01 ,  C07K2319/02

摘要： The present invention relates to a transport agent for transporting nucleic acids into eukaryotic cells and a method for producing said agent. The invention further concerns methods for transporting nucleic acids into eukaryotic cells using the transport agent according to the invention. The present invention provides an alternative transport agent and a method which are effective to allow an efficient transport of nucleic acids into eukaryotic cells. The transport agent comprises a complex forming moiety that is capable of forming complexes with at least one nucleic acid molecule and condensing said nucleic acid molecule, and at least one nuclear localization moiety comprising at least one nuclear localization signal and having an approximately neutral net charge.

摘要翻译： 本发明涉及用于将核酸输送到真核细胞中的运输剂及其制备方法。 本发明还涉及使用根据本发明的转运剂将核酸转运到真核细胞中的方法。 本发明提供了可有效地将核酸有效转运到真核细胞中的替代转运剂和方法。 转运剂包含复合物形成部分，其能够与至少一个核酸分子形成复合物并冷凝所述核酸分子，以及至少一个包含至少一个核定位信号并且具有近似中性净电荷的核定位部分。

公开(公告)号：US07320859B2

公开(公告)日：2008-01-22

申请号：US10466368

申请日：2002-01-10

申请人： Hanns-Martin Schmidt ,  Ludger Altrogge ,  Dietmar Lenz ,  Gudula Riemen ,  Helmut Brosterhus ,  Elke Lorbach ,  Juliana Helfrich ,  Katharina Hein ,  Marion Gremse ,  Tatjana Males ,  Rainer Christine ,  Gregor Siebenkotten ,  Bodo Ortmann ,  Tamara Turbanski ,  Andreas Klaes

发明人： Hanns-Martin Schmidt ,  Ludger Altrogge ,  Dietmar Lenz ,  Gudula Riemen ,  Helmut Brosterhus ,  Elke Lorbach ,  Juliana Helfrich ,  Katharina Hein ,  Marion Gremse ,  Tatjana Males ,  Rainer Christine ,  Gregor Siebenkotten ,  Bodo Ortmann ,  Tamara Turbanski ,  Andreas Klaes

IPC分类号： C12Q1/68

CPC分类号： C07K14/47 ,  A61K48/00 ,  C07K2319/035 ,  C07K2319/09 ,  C07K2319/10 ,  C07K2319/21 ,  C12N15/87

摘要： The present invention relates to a method for transfection of cells using at least one protein capable of forming nucleoprotein filaments, wherein the protein is initially modified with at least one functional component which influences one or more steps of the transfection, the nucleic acid to be transfected is then loaded with the modified protein, whereby the nucleic acid and the protein form a filament-like complex, and this complex is finally added to the cells to be transfected. The invention further relates to a transfection agent consisting of nucleoprotein filaments (NPF), with at least one nucleoprotein filament-forming protein being modified with at least one functional component for the transfection. Furthermore, the present invention relates to the use of the transfection agent according to the invention for producing a drug for gene therapeutic treatment of humans and animals. The present inventions also includes corresponding pharmaceutical preparations, especially for use in gene therapy as well as the use of such transfection agents as component in kits.

摘要翻译： 本发明涉及使用至少一种能够形成核蛋白长丝的蛋白质转染细胞的方法，其中蛋白质最初用至少一种影响转染一个或多个步骤的功能成分修饰，待转染的核酸 然后加载修饰的蛋白质，由此核酸和蛋白质形成丝状复合物，并将该复合物最终加入待转染的细胞中。 本发明还涉及由核蛋白丝（NPF）组成的转染剂，至少一种核蛋白长丝形成蛋白被至少一个用于转染的功能组分修饰。 此外，本发明涉及根据本发明的转染剂用于生产用于人和动物基因治疗的药物的用途。 本发明还包括相应的药物制剂，特别是用于基因治疗以及使用这样的转染剂作为试剂盒中的组分。

公开(公告)号：US20080220527A1

公开(公告)日：2008-09-11

申请号：US11944253

申请日：2007-11-21

申请人： Hanns-Martin SCHMIDT ,  Ludger ALTROGGE ,  Dietmar LENZ ,  Gudula RIEMEN ,  Helmut BROSTERHUS ,  Elke LORBACH ,  Juliana HELFRICH ,  Katharina HEIN ,  Marion GREMSE ,  Tatjana MALES ,  Rainer CHRISTINE ,  Gregor SIEBENKOTTEN ,  Bodo ORTMANN ,  Tamara GRABECK ,  Andrea MULLER-HARTMANN

发明人： Hanns-Martin SCHMIDT ,  Ludger ALTROGGE ,  Dietmar LENZ ,  Gudula RIEMEN ,  Helmut BROSTERHUS ,  Elke LORBACH ,  Juliana HELFRICH ,  Katharina HEIN ,  Marion GREMSE ,  Tatjana MALES ,  Rainer CHRISTINE ,  Gregor SIEBENKOTTEN ,  Bodo ORTMANN ,  Tamara GRABECK ,  Andrea MULLER-HARTMANN

IPC分类号： C12N15/00 ,  C07K14/00

CPC分类号： C07K14/47 ,  A61K48/00 ,  C07K2319/035 ,  C07K2319/09 ,  C07K2319/10 ,  C07K2319/21 ,  C12N15/87

摘要： The present invention relates to a method for transfection of cells using at least one protein capable of forming nucleoprotein filaments, wherein the protein is initially modified with at least one functional component which influences one or more steps of the transfection, the nucleic acid to be transfected is then loaded with the modified protein, whereby the nucleic acid and the protein form a filament-like complex, and this complex is finally added to the cells to be transfected. The invention further relates to a transfection agent consisting of nucleoprotein filaments (NPF), with at least one nucleoprotein filament-forming protein being modified with at least one functional component for the transfection. Furthermore, the present invention relates to the use of the transfection agent according to the invention for producing a drug for gene therapeutic treatment of humans and animals. The present inventions also includes corresponding pharmaceutical preparations, especially for use in gene therapy as well as the use of such transfection agents as component in kits.

摘要翻译： 本发明涉及使用至少一种能够形成核蛋白长丝的蛋白质转染细胞的方法，其中蛋白质最初用至少一种影响转染一个或多个步骤的功能成分修饰，待转染的核酸 然后加载修饰的蛋白质，由此核酸和蛋白质形成丝状复合物，并将该复合物最终加入待转染的细胞中。 本发明还涉及由核蛋白丝（NPF）组成的转染剂，至少一种核蛋白长丝形成蛋白被至少一个用于转染的功能组分修饰。 此外，本发明涉及根据本发明的转染剂用于生产用于人和动物基因治疗的药物的用途。 本发明还包括相应的药物制剂，特别是用于基因治疗以及使用这样的转染剂作为试剂盒中的组分。

公开(公告)号：US07999073B2

公开(公告)日：2011-08-16

申请号：US11944253

申请日：2007-11-21

申请人： Hanns-Martin Schmidt ,  Ludger Altrogge ,  Dietmar Lenz ,  Gudula Riemen ,  Helmut Brosterhus ,  Elke Lorbach ,  Juliana Helfrich ,  Katharina Hein ,  Marion Gremse ,  Tatjana Males ,  Rainer Christine ,  Gregor Siebenkotten ,  Bodo Ortmann ,  Tamara Grabek ,  Andrea Mueller-Hartmann

发明人： Hanns-Martin Schmidt ,  Ludger Altrogge ,  Dietmar Lenz ,  Gudula Riemen ,  Helmut Brosterhus ,  Elke Lorbach ,  Juliana Helfrich ,  Katharina Hein ,  Marion Gremse ,  Tatjana Males ,  Rainer Christine ,  Gregor Siebenkotten ,  Bodo Ortmann ,  Tamara Grabek ,  Andrea Mueller-Hartmann

IPC分类号： C07K14/00

CPC分类号： C07K14/47 ,  A61K48/00 ,  C07K2319/035 ,  C07K2319/09 ,  C07K2319/10 ,  C07K2319/21 ,  C12N15/87

摘要： The present invention relates to a method for transfection of cells using at least one protein capable of forming nucleoprotein filaments, wherein the protein is initially modified with at least one functional component which influences one or more steps of the transfection, the nucleic acid to be transfected is then loaded with the modified protein, whereby the nucleic acid and the protein form a filament-like complex, and this complex is finally added to the cells to be transfected. The invention further relates to a transfection agent consisting of nucleoprotein filaments (NPF), with at least one nucleoprotein filament-forming protein being modified with at least one functional component for the transfection. Furthermore, the present invention relates to the use of the transfection agent according to the invention for producing a drug for gene therapeutic treatment of humans and animals. The present inventions also includes corresponding pharmaceutical preparations, especially for use in gene therapy as well as the use of such transfection agents as component in kits.

摘要翻译： 本发明涉及使用至少一种能够形成核蛋白长丝的蛋白质转染细胞的方法，其中蛋白质最初用至少一种影响转染一个或多个步骤的功能成分修饰，待转染的核酸 然后加载修饰的蛋白质，由此核酸和蛋白质形成丝状复合物，并将该复合物最终加入待转染的细胞中。 本发明还涉及由核蛋白丝（NPF）组成的转染剂，至少一种核蛋白长丝形成蛋白被至少一个用于转染的功能组分修饰。 此外，本发明涉及根据本发明的转染剂用于生产用于人和动物基因治疗的药物的用途。 本发明还包括相应的药物制剂，特别是用于基因治疗以及使用这样的转染剂作为试剂盒中的组分。