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New therapeutic target against SMA identified

New therapeutic target against SMA identified

The inhibition of the JNK protein can slow down the progression of Spinal Muscular Athrophy or SMA, the serious neurodegenerative disorder that is the most common genetic cause of death that occurs in early childhood. This was shown in a study published in the journal Frontiers in Molecular Neuroscience by a team of Italian researchers from the Universities of Turin and Milan.

Spinal muscular atrophy (SMA) is a severe neurodegenerative disorder characterized by the loss of spinal motor neurons which causes progressive weakness, muscular atrophy and respiratory complications. Some drugs are currently being tested to stop the progression of this disease by increasing the production of SMN, the protein lacking in SMA; however, the molecular mechanisms underlying this complex disorder have not been completely clarified yet.

This study – under the leadership of Alessandro Vercelli from NICO-Neuroscience Institute Cavalieri Ottolenghi in Turin and conducted in collaboration with Tiziana Borsello from the Department of Pharmacological and Biomolecular Sciences of the University of Milano – has studied the role of the JNK protein in SMA.

In particular, researchers administered a synthetic inhibitor of the JNK activity, called D-JNKI1, in mice having SMA, and observed a slowing down of disease progression, a significant increase in motor neurons and an improvement of muscle innervation, as well as a partial, but significant, extension of survival in mice having SMA.

The study suggests that the inhibition of the JNK protein can be a future valid therapeutic strategy to slow down neurodegeneration in SMA. In additional studies, researchers from Mario Negri IRCCS have also shown that the JNK protein regulates several mechanisms associated with neurodegenerative diseases, including Alzheimer’s Disease.


Source Università Statale di Milano
Publication date 12/07/2018
Tag Health , Life Sciences