# US 2014/0155587 A1 · Antisense oligonucleotides for inducing exon skipping and methods of use thereof

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- Number: US 2014/0155587 A1
- Document ID: US20140155587A1
- Status: Published application
- Kind: A1
- Application number: 14/178,059
- Filed: 2014-02-11
- Published: 2014-06-05
- Earliest claimed: 2004-06-28
- Assignees: The University of Western Australia
- Applicants: The University of Western Australia
- Inventors: Stephen Donald WILTON; Sue Fletcher; Graham McClorey
- Web page: https://patentgrep.com/patent/US20140155587A1

## CPC

- C12N 15/113: 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)}
- A61P 21/00: Drugs for disorders of the muscular or neuromuscular system
- C12N 2310/11: Antisense
- C12N 2310/315: Phosphorothioates
- C12N 2310/321: 2'-O-R Modification
- C12N 2310/3233: Morpholino-type ring
- C12N 2310/33: of the base
- C12N 2310/3341: 5-Methylcytosine
- C12N 2310/3519: Fusion with another nucleic acid
- C12N 2320/30: Special therapeutic applications
- C12N 2320/33: Alteration of splicing
- C12N: MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA (microbiological testing media C12Q1/00)
- A61P: SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS

## Abstract

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.

## Claims

### Claim 1

(canceled)

### Claim 2

An isolated antisense oligonucleotide of 20 to 50 nucleotides in length, said oligonucleotide comprising a nucleotide sequence which is complementary to a target sequence of exon 51 of the human dystrophin pre-mRNA, wherein said target sequence comprises a nucleotide sequence that is complementary to the sequence ACA UCA AGG AAG AUG GCA UUU CUA G (SEQ ID NO: 180), and wherein said oligonucleotide comprises a morpholine ring.

### Claim 3

An isolated antisense oligonucleotide of 20 to 50 nucleotides in length, said oligonucleotide comprising a nucleotide sequence which is complementary to a target sequence of exon 51 of the human dystrophin pre-mRNA, wherein said target sequence comprises a nucleotide sequence that is complementary to the sequence ACA UCA AGG AAG AUG GCA UUU CUA G (SEQ ID NO: 180), and wherein said oligonucleotide comprises a peptide nucleic acid and/or locked nucleic acid.

### Claim 4

An isolated antisense oligonucleotide of 20 to 50 nucleotides in length, said oligonucleotide comprising a nucleotide sequence which is complementary to a target sequence of exon 51 of the human dystrophin pre-mRNA, wherein said target sequence comprises a nucleotide sequence that is complementary to the sequence ACA UCA AGG AAG AUG GCA UUU CUA G (SEQ ID NO: 180), and wherein said oligonucleotide comprises a 2′-O-methyl ribose moiety.

### Claim 5 (depends on 2)

The oligonucleotide of claim 2, wherein said oligonucleotide further comprises a phosphorodiamidate internucleoside linkage.

### Claim 6 (depends on 5)

The oligonucleotide of claim 5, wherein each linkage is a phosphorodiamidate internucleoside linkage.

### Claim 7 (depends on 6)

The oligonucleotide of claim 6, wherein the oligonucleotide is a morpholino phosphorodiamidate oligonucleotide.