5'-cap-trinucleotide- or higher oligonucleotide compounds and their uses in stabilizing RNA, expressing proteins and in therapy
The present invention relates to 5'-cap compounds, in particular the stabilization of RNA by such 5'-cap compounds, and provides compositions, such as pharmaceutical compositions, and cells comprising an RNA which is modified with such a 5 '-cap compound, as well as methods for producing a peptide or protein of interest using the compositions or cells according to the present invention. Furthermore, the present invention provides the RNA, compositions, or cells for use in therapy, in particular for use in a method of treating a disease or disorder by protein replacement therapy, genome engineering, genetic reprogramming, and immunotherapy; a method for increasing the stability of RNA in cells; a method for increasing the expression of RNA in cells; and a method for providing an RNA with a 5'-cap structure..
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Patent| Erscheinungsjahr: |
2023 |
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| Erschienen: |
2023 |
| Enthalten in: |
Europäisches Patentamt - (2023) vom: 21. Dez. Zur Gesamtaufnahme - year:2023 |
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| Sprache: |
Englisch |
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| Beteiligte Personen: |
KUHN ANDREAS [VerfasserIn] |
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| Links: |
Volltext [kostenfrei] |
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| Anmerkungen: |
Source: www.epo.org (no modifications made), First posted: 2023-12-21, Last update posted on www.tib.eu: 2024-03-04, Last updated: 2024-03-08 |
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| Patentnummer: |
AU2019235424 |
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| Förderinstitution / Projekttitel: |
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| PPN (Katalog-ID): |
EPA010515690 |
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| 245 | 1 | 0 | |a 5'-cap-trinucleotide- or higher oligonucleotide compounds and their uses in stabilizing RNA, expressing proteins and in therapy |
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| 520 | |a The present invention relates to 5'-cap compounds, in particular the stabilization of RNA by such 5'-cap compounds, and provides compositions, such as pharmaceutical compositions, and cells comprising an RNA which is modified with such a 5 '-cap compound, as well as methods for producing a peptide or protein of interest using the compositions or cells according to the present invention. Furthermore, the present invention provides the RNA, compositions, or cells for use in therapy, in particular for use in a method of treating a disease or disorder by protein replacement therapy, genome engineering, genetic reprogramming, and immunotherapy; a method for increasing the stability of RNA in cells; a method for increasing the expression of RNA in cells; and a method for providing an RNA with a 5'-cap structure. | ||
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| 650 | 4 | |a A61K: Preparations for medical, dental, or toilet purposes (devices or methods specially adapted for bringing pharmaceutical products into particular physical or administering forms a61j0003000000;chemical aspects of, or use of materials for deodorisation of air, for disinfection or sterilisation, or for bandages, dressings, absorbent pads or surgical articles a61l; soap compositions c11d) | |
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