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Antisense oligonucleotides for inducing exon skipping and methods of use thereof
US2018002697A1 · US · A1
| Field | Value |
|---|---|
| Publication number | US-2018002697-A1 |
| Application number | US-201715705172-A |
| Country | US |
| Kind code | A1 |
| Filing date | Sep 14, 2017 |
| Priority date | Jun 28, 2004 |
| Publication date | Jan 4, 2018 |
| 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 214.
First claim
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1 . (canceled) 2 . An antisense oligonucleotide of 20 to 31 bases comprising a base sequence that is 100% complementary to consecutive bases of a target region of exon 53 of the human dystrophin pre-mRNA, wherein the target region is within annealing site H53A(+23+47) and annealing site H53A(+39+69), wherein the base sequence comprises at least 12 consecutive bases of CUG AAG GUG UUC UUG UAC UUC AUC C (SEQ ID NO: 195), in which uracil bases are thymine bases, wherein the antisense oligonucleotide is a morpholino antisense oligonucleotide, and wherein the antisense oligonucleotide induces exon 53 skipping; or a pharmaceutically acceptable salt thereof. 3 . 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 a target region of exon 53 of the human dystrophin pre-mRNA, wherein the target region is within annealing site H53A(+23+47) and annealing site H53A(+39+69), wherein the base sequence comprises at least 12 consecutive bases of CUG AAG GUG UUC UUG UAC UUC AUC C (SEQ ID NO: 195), in which uracil bases are thymine bases, wherein the antisense oligonucleotide is a morpholino antisense oligonucleotide, and wherein the antisense oligonucleotide induces exon 53 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
of the base · CPC title
Phosphorothioates · CPC title
Morpholino-type ring · CPC title
2'-O-R Modification · CPC title
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- What does patent US2018002697A1 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 214.
- 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 Jan 04 2018 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).