Periodic Reporting for period 1 - Treat-HSP (Development of new therapy for rare motor neuron diseases)
Periodo di rendicontazione: 2018-11-01 al 2020-04-30
Hereditary spastic paraplegias are a group of neurodegenerative diseases characterized by spasticity and motor dysfunction and affecting about 5 persons in 100,000 in Europe. There are currently no curative treatment to prevent or slow down the progression of symptoms in these diseases. In the most frequent form of autosomal recessive hereditary spastic paraplegia due to mutation of SPG11 gene, we identified a putative therapeutic strategy that aimed at preventing the synthesis of specific lipids, gangliosides.
The ERC-POC funding allowed us to demonstrate that a method decreasing the expression of a key enzyme for the synthesis of gangliosides, GM3 synthase, slowed down the progression of symptoms and slightly decreased neuronal death in an animal model of the disease. This proof of concept allowed us to team up with a biotech to pursue this project and translate it into treatment for patients in a few years.
The ERC-POC funding allowed us to demonstrate that a method decreasing the expression of a key enzyme for the synthesis of gangliosides, GM3 synthase, slowed down the progression of symptoms and slightly decreased neuronal death in an animal model of the disease. This proof of concept allowed us to team up with a biotech to pursue this project and translate it into treatment for patients in a few years.