Harvard Group IDs new target for SMA

In a paper published in the Journal of Clinical Investigation, researchers from Harvard University show that survival motor neuron (SMN) proteins are degraded via autophagy, and suggest inhibiting the autophagy receptor sequestosome 1 (SQSTM1; p62) could increase SMN protein levels and help treat spinal muscular atrophy.

Previous studies suggested that SMN proteins were degraded primarily via the ubiquitin-proteasome system, but the role of autophagy in the protein's degradation -- known to degrade aggregated proteins in neurodegenerative diseases -- was unknown...