Motoneuron diseases are a class of neurological disorders which affect the activity and survival of motoneurons, and therefore severely impair the neuromuscular function of the affected patients.
Amyotrophic Lateral Sclerosis leads to motoneuron degeneration in adults, causing progressive paralysis. The disease is typically fatal within 2-5 years, because of respiratory failure. There is no treatment that can effectively prevent disease progression.
The PTBTG platform is active in developing gene therapy approaches to genetically modify the different cell types implicated in motoneuron diseases, in particular in the context of ALS.