Modulation of exon recognition in pre-mrna by interfering with the binding of sr proteins and by interfering with secodary rna structure
First Claim
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1. A method for generating an oligonucleotide, the method comprising:
- determining a binding site or putative binding site for an SR (Ser-Arg) protein in RNA of an exon, andproducing an oligonucleotide that is complementary to said RNA and that at least partially overlaps said binding site or putative binding site.
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Abstract
The invention provides a method for generating an oligonucleotide with which an exon may be skipped in a pre-mRNA and thus excluded from a produced mRNA thereof. Further provided are methods for altering the binding of an SR protein and/or methods for altering the secondary structure of an mRNA to interfere with splicing processes and uses of the oligonucleotides and methods in the treatment of disease. Further provided are pharmaceutical compositions and methods and means for inducing skipping of several exons in a pre-mRNA.
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24 Claims
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1. A method for generating an oligonucleotide, the method comprising:
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determining a binding site or putative binding site for an SR (Ser-Arg) protein in RNA of an exon, and producing an oligonucleotide that is complementary to said RNA and that at least partially overlaps said binding site or putative binding site. - View Dependent Claims (2, 3, 4, 5, 6, 7, 8, 9, 10, 11, 12, 13, 15, 19)
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- 14. An oligonucleotide comprising the sequence as depicted in Table 2 or an equivalent thereof.
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16. (canceled)
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17. (canceled)
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18. (canceled)
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21. A method for altering the efficiency with which an exon in a pre-mRNA is recognized by a splicing machinery, said pre-mRNA being encoded by a gene comprising at least two exons and at least one intron, said method comprising:
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providing a transcription system comprising said splicing machinery and said gene, with a first oligonucleotide selected from the group consisting of an oligonucleotide comprising the sequence as depicted in Table 2 and an oligonucleotide generated by a method comprising determining a binding site or putative binding site for an SR (Ser-Arg) protein in RNA of an exon and producing an oligonucleotide that is complementary to said RNA and that at least partially overlaps said binding site or putative binding site, wherein said first oligonucleotide is capable of hybridizing to at least one of said exons, and allowing for transcription and splicing to occur in said transcription system. - View Dependent Claims (22, 23, 24)
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Specification