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1. (WO2017001554) RÉGIONS NON TRADUITES (UTR) AUGMENTANT L'EFFICACITÉ DE TRADUCTION DES MOLÉCULES D'ARN
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CLAIMS

An RNA molecule comprising

(a) a coding region coding for a polypeptide; and

(b) upstream of said coding region one or more UTR(s) comprising the sequence as shown in SEQ ID NO:1 or a sequence which shows 1 to 4 substitutions in comparison to SEQ ID NO:1 and which results in an RNA molecule having the same or a higher translation efficiency as an RNA molecule comprising an UTR comprising SEQ ID NO:1 ; and/or

(c) downstream of said coding region one or more UTR(s) comprising the sequence as shown in SEQ ID NO:2 or a sequence which shows 1 to 7 substitutions in comparison to SEQ ID NO:2 and which results in an RNA molecule having the same or a higher translation efficiency as an RNA molecule comprising an UTR comprising SEQ ID NO:2;

wherein said polypeptide encoded by said coding region is not a cytochrome b-245 alpha polypeptide (CYBA).

The RNA molecule according to claim 1 , wherein said UTR(s) as defined in claim 1(b) is/are located at the 5' end of the coding region as defined in claim 1(a).

The RNA molecule according to claim 1 , wherein said UTR(s) as defined in claim 1(c) is/are located at the 3' end of the coding region as defined in claim 1(a).

The RNA molecule according to any one of claims 1 to 3, wherein said UTR(s) as defined in claim 1(b) is/are located at the 5' end of the coding region as defined in claim 1(a) and wherein said UTR(s) as defined in claim 1(c) is/are located at the 3' end of the coding region as defined in claim 1(a).

5. The RNA molecule according to any one of claims 1 to 4, which comprises one UTR as defined in claim 1(b) at the 5' end of the coding region as defined in claim 1(a) and which comprises two UTRs as defined in claim 1(c) at the 3' end of the coding region as defined in claim 1(a).

6. The RNA molecule according to claim 1 or 3, which comprises two UTRs as defined in claim 1(c) at the 3' end of the coding region as defined in claim 1(a).

7. The RNA molecule according to any one of claims 1 to 6, wherein the RNA molecule comprises a poly-A tail at the 3' end.

8. The RNA molecule according to any one of claims 1 to 7, wherein the poly-A tail has a length of at least 120 nucleotides.

9. A nucleic acid molecule encoding the RNA molecule of any one of claims 1 to 8.

10. A vector comprising the nucleic acid molecule of claim 9.

11. A host cell comprising the vector of claim 10.

12. A pharmaceutical composition comprising the RNA molecule according to any one of claims 1 to 8, the nucleic acid molecule according to claim 9, the vector according to claim 10 or the host cell according to claim 11 and optionally a pharmaceutically acceptable carrier.

13. The pharmaceutical composition of claim 12 for use in RNA-based therapies.

14. A kit comprising the RNA molecule according to any one of claims 1 to 8, the nucleic acid molecule according to claim 9, the vector according to claim 10 or the host cell according to claim 11.

15. Use of one or more UTR(s) as defined in claim 1(b) and/or one or more UTR(s) as defined in claim 1(c) for increasing the efficiency of translating a coding region of an RNA molecule into a polypeptide or a protein encoded by said coding region.