Blocking p62-dependent SMN degradation ameliorates spinal muscular atrophy disease phenotypes

The Journal of clinical investigation

Natalia Rodriguez-Muela, Andrey Parkhitko, Tobias Grass, …, Norbert Perrimon, Rajat Singh, Lee L Rubin

• DOI: 10.1172/jci95231
• PMID: 29672276
• PMCID: PMC6025996