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Last Updated: March 29, 2024

Details for Patent: 9,035,040


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Title:Antisense oligonucleotides for inducing exon skipping and methods of use thereof
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.
Inventor(s): Wilton; Stephen Donald (Applecross, AU), Fletcher; Sue (Bayswater, AU), McClorey; Graham (Bayswater, AU)
Assignee: The University of Western Australia (Crawley, AU)
Filing Date:Jun 27, 2014
Application Number:14/317,952
Claims:1. An antisense oligonucleotide of 25 nucleotides comprising at least 20 consecutive bases of C AUU CAA CUG UUG CCU CCG GUU CUG AAG GUG (SEQ ID NO: 193), in which cytosine bases are 5-methylcytosine bases, wherein the antisense oligonucleotide is a 2'-O-methyl phosphorothioate oligoribonucleotide, and wherein the antisense oligonucleotide specifically hybridizes to an exon 53 target region of the human dystrophin pre-mRNA to induce exon 53 skipping.

2. A pharmaceutical composition comprising an antisense oligonucleotide of 25 nucleotides comprising at least 20 consecutive bases of C AUU CAA CUG UUG CCU CCG GUU CUG AAG GUG (SEQ ID NO: 193), in which cytosine bases are 5-methylcytosine bases, wherein the antisense oligonucleotide is a 2'-O-methyl phosphorothioate oligoribonucleotide, and wherein the antisense oligonucleotide specifically hybridizes to an exon 53 target region of the human dystrophin pre-mRNA to induce exon 53 skipping, and a pharmaceutically acceptable carrier.

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