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Antisense Polynucleotides to Induce Exon Skipping and Methods of Treating Dystrophies
US2015225718A1 · US · A1
| Field | Value |
|---|---|
| Publication number | US-2015225718-A1 |
| Application number | US-201314426348-A |
| Country | US |
| Kind code | A1 |
| Filing date | Sep 6, 2013 |
| Priority date | Sep 6, 2012 |
| Publication date | Aug 13, 2015 |
| Grant date | — |
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Abstract
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Antisense polynucleotides and their use in pharmaceutical compositions to induce exon skipping in targeted exons of the gamma sarcoglycan gene are provided, along with methods of preventing or treating dystrophic diseases such as Limb-Girdle Muscular Dystrophy. One aspect the disclosure provides an isolated antisense polynucleotide wherein the polynucleotide specifically hybridizes to an exon target region of a gamma sarcoglycan RNA, wherein the exon is selected from the group consisting of exon 4 (SEQ ID NO:1), exon 5 (SEQ ID NO: 2), exon 6 (SEQ ID NO: 3), exon 7 (SEQ ID NO: 4) and a combination thereof. In some embodiments, the antisense polynucleotide cannot form an RNase H substrate, and in further embodiments the antisense polynucleotide comprises a modified polynucleotide backbone.
First claim
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What is claimed is: 1 . An isolated antisense polynucleotide wherein the polynucleotide specifically hybridizes to an exon target region of a gamma sarcoglycan RNA, wherein the exon is selected from the group consisting of exon 4 (SEQ ID NO: 1), exon 5 (SEQ ID NO: 2), exon 6 (SEQ ID NO: 3), exon 7 (SEQ ID NO: 4) and a combination thereof. 2 . The antisense polynucleotides of claim 1 , wherein the polynucleotide cannot form an RNase H substrate.…
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Human Necessities · mapped topic
Chemistry & Metallurgy · mapped topic
Chemistry & Metallurgy · mapped topic
Chemistry & Metallurgy · mapped topic
Chemistry & Metallurgy · mapped topic
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- What does patent US2015225718A1 cover?
- Antisense polynucleotides and their use in pharmaceutical compositions to induce exon skipping in targeted exons of the gamma sarcoglycan gene are provided, along with methods of preventing or treating dystrophic diseases such as Limb-Girdle Muscular Dystrophy. One aspect the disclosure provides an isolated antisense polynucleotide wherein the polynucleotide specifically hybridizes to an exon t…
- Who is the assignee on this patent?
- Univ Chicago
- 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 Thu Aug 13 2015 00:00:00 GMT+0000 (Coordinated Universal Time) (A1). 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).