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Modular transfection systems based on nucleoprotein filaments

机译:基于核蛋白丝的模块化转染系统

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.
机译:本发明涉及一种使用至少一种能够形成核蛋白细丝的蛋白质转染细胞的方法,其中该蛋白质首先被至少一种影响转染一个或多个步骤的功能性成分修饰,该功能被转染的核酸。然后将修饰的蛋白质上样到RNA上,由此核酸和蛋白质形成丝状复合物,并且最终将该复合物添加到待转染的细胞中。本发明进一步涉及由核蛋白丝(NPF)组成的转染剂,其中至少一种核蛋白丝形成蛋白被至少一种用于转染的功能性成分修饰。此外,本发明涉及根据本发明的转染剂在生产用于人和动物的基因治疗的药物中的用途。本发明还包括相应的药物制剂,特别是用于基因治疗的药物制剂,以及这种转染剂作为试剂盒中的组分的用途。

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