Combining Two New Antisense Oligonucleotides Increases SMN Protein Levels in SMA Mice, Study Shows
Researchers have shown that the technology called antisense oligonucleotides, used in the only FDA-approved therapy for SMA, Spinraza (nusinersen), can be improved upon. They designed two new sequences that significantly increased SMN production in human cells and in a mouse model of SMA when administered alone or combined. The…