Fratta lab

Molecular Neurodegeneration Laboratory

Our lab aims to identify and understand molecular mechanisms underlying motor neuron diseases (MND) and use these to develop novel therapeutic strategies. 

One of our main areas of research is to investigate what happens when certain RNA-binding proteins cluster together in cases of MND. 
 
We use tools including stem cell-derived neurons, mouse models and post-mortem material to study this disease. We look at how changes specific to the disease can disrupt the normal functions of RNA, and whether these disruptions are related to the symptoms of the disease. We can use our findings to develop new gene therapy approaches to correct these disruptions in disease-affected cells.

Part of our work involves looking at a specific type of MND called Kennedy’s disease. Kennedy’s disease is caused by irregularities in a specific gene leading to muscle weakness primarily in men. We're interested in understanding what factors determine the likelihood and severity of the disease and whether there is any way to safely treat it by inactivating the affected gene in muscle tissue.
 
We're also working on finding ways to detect MND early using specific markers, known as biomarkers, in bodily fluids.