abstract |
Compositions and methods are provided for the inhibition, treatment and/or prevention of degenerative myelopathy (DM) or amyotrophic lateral sclerosis (ALS). In accordance with the instant invention, nucleic acid molecules for inhibiting the expression of the superoxide dismutase 1 gene (SOD) are provided. In a particular embodiment, the nucleic acid molecules comprise an annealing domain operably linked to at least one effector domain, wherein the annealing domain hybridizes to the pre-mRNA of SOD (e.g., canine or human) and wherein the effector domain hybridizes to the U1 snRNA of U1 snRNP. |