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MODULAR TRANSFECTION SYSTEMS BASED ON NUCLEOPROTEIN FILAMENTS

机译:基于核蛋白长丝的模块化翻译系统

摘要

The invention relates to a method for the transfection of cells using at least one protein capable of forming nucleoprotein filaments. Said protein is first modified by at least one functional constituent which influences at least one step of the transfection, and the nucleic acid to be transfected is then loaded with the modified protein. The nucleic acid and the protein form a filamentary complex, and said complex is then added to the cells to be transfected. The invention also relates to a transfection agent of nucleoprotein filaments (NPF), at least one protein forming a nucleoprotein filament being modified by at least one functional constituent for the transfection. The invention further relates to the use of the inventive transfection agent for producing a medicament for the gene therapeutic treatment of humans and animals. Finally, the invention relates to corresponding pharmaceutical preparations, especially for use in gene therapy, and the use of such transfection agents in the form of constituents in kits. Fig. 20. 1. DNA 2. protein building blocks 3. charged DNA 4. functions 5. association with the cell surface 6. passage through the membrane 7. emergence from the endosomes 8. transport into the nucleus 9. other function 10. cell membrane 11. nucleus membrane 12. separation in nucleus 13. expression
机译:本发明涉及一种使用至少一种能够形成核蛋白丝的蛋白质转染细胞的方法。所述蛋白质首先被影响至少一个转染步骤的至少一种功能性成分修饰,然后将修饰的蛋白质装载到待转染的核酸上。核酸和蛋白质形成丝状复合物,然后将所述复合物添加到待转染的细胞中。本发明还涉及核蛋白丝(NPF)的转染剂,形成核蛋白丝的至少一种蛋白被至少一种用于转染的功能性成分修饰。本发明还涉及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。最后,本发明涉及相应的药物制剂,特别是用于基因治疗的药物制剂,以及此类转染剂以试剂盒中成分的形式的用途。图20. 1. DNA 2.蛋白质构件3.带电的DNA 4.功能5.与细胞表面结合6.穿过膜7.从内体出来8.运入细胞核9.其他功能10。细胞膜11.细胞核膜12.细胞核分离13.表达

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