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Increasing SMN Protein May Be Therapeutic Even After SMA Disease Onset

Congratulations to Umrao Monani, Assistant Professor at the Motor Neuron Center of Columbia University and Cathleen Lutz, Associate Director of Genetic Research Science at The Jackson Laboratory, and their colleagues for their new publication “Postsymptomatic Restoration of SMN Rescues the Disease Phenotype in a Mouse Model of Severe Spinal Muscular Atrophy” in the Journal of Clinical Investigation on July 25, 2011. Using a new mouse model, these investigators discovered that restoring levels of SMN protein after disease onset extends lifespan and improves motor function. These results raise the possibility that an SMN-mediated treatment may be effective in patients with SMA even if administered at later stages of the disease. The discovery was made by generating mice harboring an inducible SMN rescue allele that allows SMN expression to be turned on at different time points during the course of the disease. To view the article, please visit http://www.ncbi.nlm.nih.gov/pubmed/21785219.

This study was supported by the SMA Foundation.