Oligomeric nucleic acid molecule and application thereof

Abstract

The present invention relates to a small activating nucleic acid molecule for treating spinal muscular atrophy and use thereof. The small activating nucleic acid molecule comprises a sense nucleic acid strand and an antisense nucleic acid strand, wherein the sense nucleic acid strand and the antisense nucleic acid strand are independently an oligonucleotide strand of 16 to 35 nucleotides in length, in which one nucleotide strand has at least 75% base homology or complementarity to a target selected from a promoter region of a target gene SMN2. The present invention also relates to a pharmaceutical composition comprising the small activating nucleic acid molecule disclosed herein and optionally, a pharmaceutically acceptable carrier, and a method for up-regulating the expression of a target gene in cells and methods for treating a disease induced by insufficient expression of a target gene with the small activating nucleic acid molecule or the pharmaceutical composition comprising the small activating nucleic acid molecule disclosed herein.

Dependencies are inferred from the claim text.