Patent ID: 8802642
Filing Date: 2014-08-12
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 isolated oligonucleotide reagent comprising a nucleotide sequence at least 90% complementary over its entire length to a target region of the sequence 5′-CUGGUGUCCACAGAGGAC-3 (SEQ ID NO:3), wherein said oligonucleotide contains between about 10 nucleotide bases and about 20 nucleotide bases, and said nucleotide sequence is effective to hybridize to at least 10 contiguous bases of said target region, such that the level of exon 7-containing SMN2 mRNA relative to exon-deleted SMN2 mRNA in the cell or cell extract is enhanced.