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Antisense oligonucleotides for inducing exon skipping and methods of use thereof
US2015376615A1 · US · A1
| Field | Value |
|---|---|
| Publication number | US-2015376615-A1 |
| Application number | US-201514852090-A |
| Country | US |
| Kind code | A1 |
| Filing date | Sep 11, 2015 |
| Priority date | Jun 28, 2004 |
| Publication date | Dec 31, 2015 |
| Grant date | — |
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Abstract
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An antisense molecule capable of binding to a selected target site to induce exon skipping in the dystrophin gene, as set forth in SEQ ID NO: 1 to 202.
First claim
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1 .- 20 . (canceled) 21 . An antisense oligonucleotide of 20 to 31 bases comprising a base sequence that is 100% complementary to consecutive bases of the human dystrophin pre-mRNA, wherein the base sequence comprises at least 20 consecutive bases of GGG AUC CAG UAU ACU UAC AGG CUC C (SEQ ID NO: 175), in which uracil bases are thymine bases, wherein the antisense oligonucleotide is a morpholino antisense oligonucleotide, and wherein the antisense oligonucleotide induces exon 50 skipping; or a pharmaceutically acceptable salt thereof. 22 . A pharmaceutical composition comprising: (i) an antisense oligonucleotide of 20 to 31 bases comprising a base sequence that is 100% complementary to consecutive bases of the human dystrophin pre-mRNA, wherein the base sequence comprises at least 20 consecutive bases of GGG AUC CAG UAU ACU UAC AGG CUC C (SEQ ID NO: 175), in which uracil bases are thymine bases, wherein the antisense oligonucleotide is a morpholino antisense oligonucleotide, and wherein the antisense oligonucleotide induces exon 50 skipping, or a pharmaceutically acceptable salt thereof; and (ii) a pharmaceutically acceptable carrier.
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Drugs for disorders of the muscular or neuromuscular system · CPC title
Alteration of splicing · CPC title
Fusion with another nucleic acid · CPC title
- C12N15/113Primary
Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; {Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing (when used in plants C12N15/8218)} · CPC title
Antisense · CPC title
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- What does patent US2015376615A1 cover?
- An antisense molecule capable of binding to a selected target site to induce exon skipping in the dystrophin gene, as set forth in SEQ ID NO: 1 to 202.
- Who is the assignee on this patent?
- Univ Western Australia
- 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 Dec 31 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).