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Modulation of exon recognition in pre-mRNA by interfering with the secondary RNA structure
US9896687B2 · US · B2
| Field | Value |
|---|---|
| Publication number | US-9896687-B2 |
| Application number | US-23349505-A |
| Country | US |
| Kind code | B2 |
| Filing date | Sep 21, 2005 |
| Priority date | Mar 21, 2003 |
| Publication date | Feb 20, 2018 |
| Grant date | Feb 20, 2018 |
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- Title
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Abstract
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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 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.
First claim
Opening claim text (preview).
What is claimed is: 1. An isolated antisense oligonucleotide of 18 nucleotides consisting of the base sequence of the sequence cuguugccuccgguucug (SEQ ID NO: 29), wherein said oligonucleotide induces exon 53 skipping in the human dystrophin pre-mRNA, said oligonucleotide comprising a modification selected from the group consisting of: 2′-O- methyl, 2′-O-methylphosphorothioate, a morpholine ring, a phosphorodiamidate linkage, a peptide nucleic acid and a locked nucleic acid.
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Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00 · CPC title
for myasthenia gravis · CPC title
Drugs for disorders of the muscular or neuromuscular system · CPC title
having an additional ring, e.g. LNA, ENA · CPC title
2'-O-R Modification · CPC title
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- What does patent US9896687B2 cover?
- 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 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 composition…
- Who is the assignee on this patent?
- Van Ommen Garrit Jan Boudewijn, Van Deutekom Judith Christina Theodora, Den Dunnen Johannes Theodorus, and 2 more
- What technology area does this patent fall under?
- Primary CPC classification C12N15/113. Mapped technology areas include Chemistry & Metallurgy.
- When was this patent published?
- Publication date Tue Feb 20 2018 00:00:00 GMT+0000 (Coordinated Universal Time) (B2). Legal status and post-grant events are not shown on this page.
- What related patents are in patentsdb?
- We list 8 related publications on this page (citations in our corpus or others sharing the same primary CPC).