Patent ID: 8586560

Claim:
A method for restoring the function of a mutated dysferlin in a subject comprising the step of preventing splicing of one or more exons which encode amino acid sequences that cause said dysferlin dysfunction by providing said subject one or more antisense oligonucleotides complementary to nucleic acid sequences that are necessary for correct splicing of said one or more exons which encode amino acid sequences that cause said dysferlin dysfunction, said one or more antisense oligonucleotides being provided in an amount sufficient to cause skipping of said one or more exons during splicing, wherein said one or more antisense oligonucleotides are complementary to nucleic acid sequences that are necessary for correct splicing of a region comprising exon 32 of the DYSF gene, and wherein said one or more antisense oligonucleotides are complementary to the nucleic acid sequence SEQ ID NO:1 or SEQ ID NO:2.