Patent ID: 9217147
Filing Date: 2015-12-22
Classification: A61K,A61P,C12N

Abstract:
1. A method of enhancing the level of exon 7-containing SMN2 mRNA relative to exon-deleted SMN2 mRNA in a cell or cell extract, comprising contacting the cell or cell extract with an oligonucleotide of 8 to 19 nucleotides in length which is complementary to at least 8 contiguous nucleotides of CUGCCAGCAUUAUGAAAG (nucleotides 7 to 24 of SEQ ID NO: 2) of intron 7 of the SMN2 gene, wherein said oligonucleotide is complementary to nucleotide 10 ( 10 C) and; said oligonucleotide is 8 to 14 nucleotides in length and is complementary to nucleotides 7, 8, and 9 of intron 7 of the SMN2 gene; or said oligonucleotide is 8 to 14 nucleotides in length and is complementary to nucleotides 8 and 9 of intron 7 of the SMN2 gene; or said oligonucleotide is 11, 13, 14, 16, 17, or 19 nucleotides in length and is complementary to none of nucleotides 7, 8, and 9; or said oligonucleotide is 11 to 14 nucleotides in length and is complementary to nucleotide 9 of intron 7 of the SMN2 gene; such that the level of exon 7-containing SMN2 mRNA relative to exon-deleted SMN2 mRNA in the cell or cell extract is enhanced.