Compounds for the modulation of smn2 splicing

摘要

The present invention relates to oligomer compounds (oligomers) which target nucleic acids encoding human SMN2 in a cell, leading to modulation of SMN2 mRNA splicing which favors full length SMN2 mRNA rather than the poorly functional truncated transcript, SMN2Δ7. Reduction of SMNA7 mRNA expression and/or increase in full length SMN2 mRNA expression are beneficial for the treatment of diseases or disorders associated with overexpression or undesirably high levels of aberrant forms of SMN2, particularly SMN2Δ7, such as spinal muscular atrophy (SMA).